PNA-mediated alteration of mRNA splicing (316 views)
Minerva Biotecnol (ISSN: 1120-4826), 2008; 20(4): 175-181.
Export to BibTeX Export to EndNote
Paper type: Journal Article,
Impact factor: 0.1, 5-year impact factor: 0.211
Url: http://www.scopus.com/inward/record.url?eid=2-s2.0-65449172851&partnerID=40&md5=98748f6c4302d0b31e072537f82a7ed5
Keywords: β-Thalassemia, Peptide Nucleic Acids, Splicing, Beta Globin, Messenger Rna, Protein Bcl X, Antisense Vector, Article, Beta Thalassemia, Cellular Distribution, Dna Modification, Gene Targeting, Genetic Transduction, Human, Nonhuman, Nonviral Gene Delivery System, Rna Interference, Rna Sequence, Rna Splicing, Sequence Analysis, ß-Thalassemia,
Affiliations:
*** IBB - CNR ***
BioPharmaNet, Department of Biochemistry and Molecular Biology, University of Ferrara, via Fossato di Mortara 74, 44100 Ferrara, Italy
Laboratory for the Development of Pharmacological and Pharmacogenomic Therapy of Thalassaemia, Biotechnology Center, University of Ferrara, Ferrara, Italy
Institute of Biostructure and Bioimaging, University of Naples, Naples, Italy
School of Biotechnological Sciences, University of Naples Federico II, Naples, Italy
References:
Nielsen, P.E., Egholm, M., Berg, R.H., Buchardt, O., Sequence selective recognition of DNA by strand displacement with a thymine-substituted polyamide (1991) Science, 254, pp. 1497-150
Lundin, K.E., Good, L., Strömberg, R., Gräslund, A., Smith, C.I., Biological activity and biotechnological aspects of peptide nucleic acid (2006) Adv Genet, 56, pp. 1-51
Nielsen, E., RNA targeting using peptide nucleic acid (2006) Handb Exp Pharmacol, 173, pp. 395-403
Yin, H., Lu, Q., Wood, M., Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice (2008) Mol Ther, 16, pp. 38-45
Romanelli, A., Pensato, S., Di Niola, E., Feriotto, G., Breveglieri, G., Zaccaro, L., Antisense PNA and PNA-peptide conjugates for the modulation of the β-globin gene splicing (2005) Biopolymers, 80, pp. 551-552
Nielsen, P.E., Gene targeting using peptide nucleic acid (2005) Methods Mol Biol, 288, pp. 343-358
Tonelli, R., Purgato, S., Camerin, C., Fronza, R., Bologna, F., Alboresi, S., Anti-gene peptide nucleic acid specifically inhibits MYCN expression in human neuroblastoma cells leading to cell growth inhibition and apoptosis (2005) Mol Cancer Ther, 4, pp. 779-786
Pesce, C.D., Bolacchi, F., Bongiovanni, B., Cisotta, F., Capozzi, M., Diviacco, S., Anti-gene peptide nucleic acid targeted to proviral HIV-1 DNA inhibits in vitro HIV-1 replication (2005) Antiviral Res, 66, pp. 13-22
Corradini, R., Feriotto, G., Sforza, S., Marchelli, R., Gambari, R., Enhanced recognition of cystic fibrosis W1282X DNA point mutation by chiral peptide nucleic acid probes by a surface plasmon resonance biosensor (2004) J Mol Recognit, 17, pp. 76-84
Feriotto, G., Corradini, R., Sforza, S., Bianchi, N., Mischiati, C., Marchelli, R., Peptide nucleic acids and biosensor technology for realtime detection of the cystic fibrosis W1282X mutation by surface plasmon resonance (2001) Lab Invest, 81, pp. 1415-1427
Ohtsuki, T., Fujimoto, T., Kamimukai, M., Kumano, C., Kitamatsu, M., Sisido, M., Isolation of small RNAs using biotinylated PNAs (2008) J Biochem, 144, pp. 415-418
Bonham, M.A., Brown, S., Boyd, A.L., Brown, P.H., Bruckenstein, D.A., Hanvey, J.C., An assessment of the antisense properties of RNase H-competent and steric-blocking oligomers (1995) Nucleic Acids Res, 23, pp. 1197-1203
Weatherall, D.J., Clegg, J.B., (2001) The thalassemia syndromes, pp. 133-191. , Oxford: Blackwell Scientific
Ho, P.J., The regulation of beta globin gene expression and beta thalassemia (1999) Pathology, 31, pp. 315-324
Eleftheriou, A., The pathophysiology of thalassaemia: Lack of β-chains and excess of α-chains (2007) About Thalassemia, pp. 16-18. , Eleftheriou A, editor, Nicosia: The Thalassemia International Federation publisher
Schrier, S.L., Pathophysiology of thalassemia (2002) Curr Opin Hematol, 9, pp. 123-126
Bank, A., Regulation of human fetal hemoglobin: New players, new complexities (2006) Blood, 107, pp. 435-443
Voon, H.P., Wardan, H., Vadolas, J., Co-inheritance of alpha- and beta-thalassaemia in mice ameliorates thalassaemic phenotype (2007) Blood Cells Mol Dis, 39, pp. 184-188
Gambari, R., Fibach, E., Medicinal chemistry of fetal hemoglobin inducers for treatment of beta-thalassemia (2007) Curr Med Chem, 14, pp. 199-212
Xie, S.Y., Ren, Z.R., Zhang, J.Z., Guo, X.B., Wang, Q.X., Wang, S., Restoration of the balanced alpha/beta-globin gene expression in beta654-thalassemia mice using combined RNAi and antisense RNA approach (2007) Hum Mol Genet, 16, pp. 2616-2625
Bianchi, N., Borgatti, M., Gambari, R., Gene Silencing in thalassemia (2008) Minerva Biotecnologica, 20, pp. 51-55
Abes, S., Ivanova, G.D., Abes, R., Arzumanov, A.A., Williams, D., Owen, D., Peptide-based delivery of steric-block PNA oligonucleotides (2009) Methods Mol Biol, 480, pp. 85-99
Lundin, P., Johansson, H., Guterstam, P., Holm, T., Hansen, M., Langel, U., Distinct uptake routes of cell-penetrating peptide conjugates (2008) Bioconjug Chem, 19, pp. 2535-2542
Ivanova, G.D., Arzumanov, A., Abes, R., Yin, H., Wood, M.J., Lebleu, B., Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle (2008) Nucleic Acids Res, 36, pp. 6418-6428
Ivanova, G.D., Fabani, M.M., Arzumanov, A.A., Abes, R., Yin, H., Lebleu, B., PNA-peptide conjugates as intracellular gene control agents (2008) Nucleic Acids Symp Ser (Oxf), 52, pp. 31-32
Wright, D.G., Zhang, Y., Murphy, J.R., Effective delivery of antisense peptide nucleic acid oligomers into cells by anthrax protective antigen (2008) Biochem Biophys Res Commun, 376, pp. 200-205
Shiraishi, T., Hamzavi, R., Nielsen, P.E., Subnanomolar antisense activity of phosphonate-peptide nucleic acid (PNA) conjugates delivered by cationic lipids to HeLa cells (2008) Nucleic Acids Res, 36, pp. 4424-4432
Robertson, K.L., Yu, L., Armitage, B.A., Lopez, A.J., Peteanu, L.A., Fluorescent PNA probes as hybridization labels for biological RNA (2006) Biochemistry, 45, pp. 6066-6074
Rasmussen, F.W., Bendifallah, N., Zachar, V., Shiraishi, T., Fink, T., Ebbesen, P., Evaluation of transfection protocols for unmodified and modified peptide nucleic acid (PNA) oligomers (2006) Oligonucleotides, 16, pp. 43-57
Albertshofer, K., Siwkowski, A.M., Wancewicz, E.V., Esau, C.C., Watanabe, T., Nishihara, K.C., Structure-activity relationship study on a simple cationic peptide motif for cellular delivery of antisense peptide nucleic acid (2005) J Med Chem, 48, pp. 6741-6749
Karras, J.G., Maier, M.A., Lu, T., Watt, A., Manoharan, M., Peptide nucleic acids are potent modulators of endogenous pre-mRNA splicing of the murine interleukin-5 receptor-alpha chain (2001) Biochemistry, 40, pp. 7853-7859
Siwkowski, A.M., Malik, L., Esau, C.C., Maier, M.A., Wancewicz, E.V., Albertshofer, K., Identification and functional validation of PNAs that inhibit murine CD40 expression by redirection of splicing (2004) Nucleic Acids Res, 32, pp. 2695-2706
Wilusz, J.E., Devanney, S.C., Caputi, M., Chimeric peptide nucleic acid compounds modulate splicing of the bcl-x gene in vitro and in vivo (2005) Nucleic Acids Res, 33, pp. 6547-6554
El-Andaloussi, S., Johansson, H.J., Lundberg, P., Langel, U., Induction of splice correction by cell-penetrating peptide nucleic acids (2006) J Gene Med, 8, pp. 1262-1273
van Vliet, L., de Winter, C.L., van Deutekom, J.C., van Ommen, G.J., Aartsma-Rus, A., Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy (2008) BMC Med Genet, 9, pp. 105-111
Gao, Y., Zu, T., Low, W.C., Orr, H.T., McIvor, R.S., Antisense RNA sequences modulating the ataxin-1 message: Molecular model of gene therapy for spinocerebellar ataxia type 1, a dominant-acting unstable trinucleotide repeat disease (2008) Cell Transplant, 17, pp. 723-734
Davis, R.L., Homer, V.M., George, P.M., Brennan, S.O., A deep intronic mutation in FGB creates a consensus exonic splicing enhancer motif that results in afibrinogenemia caused by aberrant mRNA splicing, which can be corrected in vitro with antisense oligonucleotide treatment (2009) Hum Mutat, 30, pp. 221-227
Madsen, E.C., Morcos, P.A., Mendelsohn, B.A., Gitlin, J.D., In vivo correction of a Menkes disease model using antisense oligonucleotides (2008) Proc Natl Acad Sci USA, 105, pp. 3909-3914
Mascellani, N., Tagliavini, L., Gamberoni, G., Rossi, S., Marchesini, J., Taccioli, C., Using miRNA expression data for the study of human cancer (2008) Minerva Biotecnologica, 20, pp. 23-30
Habbe, N., Koorstra, J.B., Mendell, J.T., Offerhaus, G.J., Ryu, J.K., Feldmann, G., MicroRNA miR-155 is a biomarker of early pancreatic neoplasia (2009) Cancer Biol Ther, 8, pp. 25-31
Duisters, R.F., Tijsen, A.J., Schroen, B., Leenders, J.J., Lentink, V., van der Made, I., miR-133 and miR-30 regulate connective tissue growth factor: Implications for a role of microRNAs in myocardial matrix remodeling (2009) Circ Res, 104, pp. 170-178
Chen, J.F., Callis, T.E., Wang, D.Z., microRNAs and muscle disorders (2009) J Cell Sci, 122, pp. 13-20
Xiao, B., Guo, J., Miao, Y., Jiang, Z., Huan, R., Zhang, Y., Detection of miR-106a in gastric carcinoma and its clinical significance (2009) Clin Chim Acta, 400, pp. 97-102
Abes, S., Turner, J.J., Ivanova, G.D., Owen, D., Williams, D., Arzumanov, A., Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide (2007) Nucleic Acids Res, 35, pp. 4495-4502
Nielsen, P. E., Egholm, M., Berg, R. H., Buchardt, O., Sequence selective recognition of DNA by strand displacement with a thymine-substituted polyamide (1991) Science, 254, pp. 1497-150
Lundin, K. E., Good, L., Str mberg, R., Gr slund, A., Smith, C. I., Biological activity and biotechnological aspects of peptide nucleic acid (2006) Adv Genet, 56, pp. 1-51
Nielsen, P. E., Gene targeting using peptide nucleic acid (2005) Methods Mol Biol, 288, pp. 343-358
Pesce, C. D., Bolacchi, F., Bongiovanni, B., Cisotta, F., Capozzi, M., Diviacco, S., Anti-gene peptide nucleic acid targeted to proviral HIV-1 DNA inhibits in vitro HIV-1 replication (2005) Antiviral Res, 66, pp. 13-22
Bonham, M. A., Brown, S., Boyd, A. L., Brown, P. H., Bruckenstein, D. A., Hanvey, J. C., An assessment of the antisense properties of RNase H-competent and steric-blocking oligomers (1995) Nucleic Acids Res, 23, pp. 1197-1203
Weatherall, D. J., Clegg, J. B., (2001) The thalassemia syndromes, pp. 133-191. , Oxford: Blackwell Scientific
Ho, P. J., The regulation of beta globin gene expression and beta thalassemia (1999) Pathology, 31, pp. 315-324
Schrier, S. L., Pathophysiology of thalassemia (2002) Curr Opin Hematol, 9, pp. 123-126
Voon, H. P., Wardan, H., Vadolas, J., Co-inheritance of alpha- and beta-thalassaemia in mice ameliorates thalassaemic phenotype (2007) Blood Cells Mol Dis, 39, pp. 184-188
Xie, S. Y., Ren, Z. R., Zhang, J. Z., Guo, X. B., Wang, Q. X., Wang, S., Restoration of the balanced alpha/beta-globin gene expression in beta654-thalassemia mice using combined RNAi and antisense RNA approach (2007) Hum Mol Genet, 16, pp. 2616-2625
Ivanova, G. D., Arzumanov, A., Abes, R., Yin, H., Wood, M. J., Lebleu, B., Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle (2008) Nucleic Acids Res, 36, pp. 6418-6428
Ivanova, G. D., Fabani, M. M., Arzumanov, A. A., Abes, R., Yin, H., Lebleu, B., PNA-peptide conjugates as intracellular gene control agents (2008) Nucleic Acids Symp Ser (Oxf), 52, pp. 31-32
Wright, D. G., Zhang, Y., Murphy, J. R., Effective delivery of antisense peptide nucleic acid oligomers into cells by anthrax protective antigen (2008) Biochem Biophys Res Commun, 376, pp. 200-205
Robertson, K. L., Yu, L., Armitage, B. A., Lopez, A. J., Peteanu, L. A., Fluorescent PNA probes as hybridization labels for biological RNA (2006) Biochemistry, 45, pp. 6066-6074
Rasmussen, F. W., Bendifallah, N., Zachar, V., Shiraishi, T., Fink, T., Ebbesen, P., Evaluation of transfection protocols for unmodified and modified peptide nucleic acid (PNA) oligomers (2006) Oligonucleotides, 16, pp. 43-57
Karras, J. G., Maier, M. A., Lu, T., Watt, A., Manoharan, M., Peptide nucleic acids are potent modulators of endogenous pre-mRNA splicing of the murine interleukin-5 receptor-alpha chain (2001) Biochemistry, 40, pp. 7853-7859
Siwkowski, A. M., Malik, L., Esau, C. C., Maier, M. A., Wancewicz, E. V., Albertshofer, K., Identification and functional validation of PNAs that inhibit murine CD40 expression by redirection of splicing (2004) Nucleic Acids Res, 32, pp. 2695-2706
Wilusz, J. E., Devanney, S. C., Caputi, M., Chimeric peptide nucleic acid compounds modulate splicing of the bcl-x gene in vitro and in vivo (2005) Nucleic Acids Res, 33, pp. 6547-6554
Davis, R. L., Homer, V. M., George, P. M., Brennan, S. O., A deep intronic mutation in FGB creates a consensus exonic splicing enhancer motif that results in afibrinogenemia caused by aberrant mRNA splicing, which can be corrected in vitro with antisense oligonucleotide treatment (2009) Hum Mutat, 30, pp. 221-227
Madsen, E. C., Morcos, P. A., Mendelsohn, B. A., Gitlin, J. D., In vivo correction of a Menkes disease model using antisense oligonucleotides (2008) Proc Natl Acad Sci USA, 105, pp. 3909-3914
Duisters, R. F., Tijsen, A. J., Schroen, B., Leenders, J. J., Lentink, V., van der Made, I., miR-133 and miR-30 regulate connective tissue growth factor: Implications for a role of microRNAs in myocardial matrix remodeling (2009) Circ Res, 104, pp. 170-178
Chen, J. F., Callis, T. E., Wang, D. Z., microRNAs and muscle disorders (2009) J Cell Sci, 122, pp. 13-20
Minerva Biotecnol (ISSN: 1120-4826), 2008; 20(4): 175-181.
Export to BibTeX Export to EndNote
Paper type: Journal Article,
Impact factor: 0.1, 5-year impact factor: 0.211
Url: http://www.scopus.com/inward/record.url?eid=2-s2.0-65449172851&partnerID=40&md5=98748f6c4302d0b31e072537f82a7ed5
Keywords: β-Thalassemia, Peptide Nucleic Acids, Splicing, Beta Globin, Messenger Rna, Protein Bcl X, Antisense Vector, Article, Beta Thalassemia, Cellular Distribution, Dna Modification, Gene Targeting, Genetic Transduction, Human, Nonhuman, Nonviral Gene Delivery System, Rna Interference, Rna Sequence, Rna Splicing, Sequence Analysis, ß-Thalassemia,
Affiliations:
*** IBB - CNR ***
BioPharmaNet, Department of Biochemistry and Molecular Biology, University of Ferrara, via Fossato di Mortara 74, 44100 Ferrara, Italy
Laboratory for the Development of Pharmacological and Pharmacogenomic Therapy of Thalassaemia, Biotechnology Center, University of Ferrara, Ferrara, Italy
Institute of Biostructure and Bioimaging, University of Naples, Naples, Italy
School of Biotechnological Sciences, University of Naples Federico II, Naples, Italy
References:
Nielsen, P.E., Egholm, M., Berg, R.H., Buchardt, O., Sequence selective recognition of DNA by strand displacement with a thymine-substituted polyamide (1991) Science, 254, pp. 1497-150
Lundin, K.E., Good, L., Strömberg, R., Gräslund, A., Smith, C.I., Biological activity and biotechnological aspects of peptide nucleic acid (2006) Adv Genet, 56, pp. 1-51
Nielsen, E., RNA targeting using peptide nucleic acid (2006) Handb Exp Pharmacol, 173, pp. 395-403
Yin, H., Lu, Q., Wood, M., Effective exon skipping and restoration of dystrophin expression by peptide nucleic acid antisense oligonucleotides in mdx mice (2008) Mol Ther, 16, pp. 38-45
Romanelli, A., Pensato, S., Di Niola, E., Feriotto, G., Breveglieri, G., Zaccaro, L., Antisense PNA and PNA-peptide conjugates for the modulation of the β-globin gene splicing (2005) Biopolymers, 80, pp. 551-552
Nielsen, P.E., Gene targeting using peptide nucleic acid (2005) Methods Mol Biol, 288, pp. 343-358
Tonelli, R., Purgato, S., Camerin, C., Fronza, R., Bologna, F., Alboresi, S., Anti-gene peptide nucleic acid specifically inhibits MYCN expression in human neuroblastoma cells leading to cell growth inhibition and apoptosis (2005) Mol Cancer Ther, 4, pp. 779-786
Pesce, C.D., Bolacchi, F., Bongiovanni, B., Cisotta, F., Capozzi, M., Diviacco, S., Anti-gene peptide nucleic acid targeted to proviral HIV-1 DNA inhibits in vitro HIV-1 replication (2005) Antiviral Res, 66, pp. 13-22
Corradini, R., Feriotto, G., Sforza, S., Marchelli, R., Gambari, R., Enhanced recognition of cystic fibrosis W1282X DNA point mutation by chiral peptide nucleic acid probes by a surface plasmon resonance biosensor (2004) J Mol Recognit, 17, pp. 76-84
Feriotto, G., Corradini, R., Sforza, S., Bianchi, N., Mischiati, C., Marchelli, R., Peptide nucleic acids and biosensor technology for realtime detection of the cystic fibrosis W1282X mutation by surface plasmon resonance (2001) Lab Invest, 81, pp. 1415-1427
Ohtsuki, T., Fujimoto, T., Kamimukai, M., Kumano, C., Kitamatsu, M., Sisido, M., Isolation of small RNAs using biotinylated PNAs (2008) J Biochem, 144, pp. 415-418
Bonham, M.A., Brown, S., Boyd, A.L., Brown, P.H., Bruckenstein, D.A., Hanvey, J.C., An assessment of the antisense properties of RNase H-competent and steric-blocking oligomers (1995) Nucleic Acids Res, 23, pp. 1197-1203
Weatherall, D.J., Clegg, J.B., (2001) The thalassemia syndromes, pp. 133-191. , Oxford: Blackwell Scientific
Ho, P.J., The regulation of beta globin gene expression and beta thalassemia (1999) Pathology, 31, pp. 315-324
Eleftheriou, A., The pathophysiology of thalassaemia: Lack of β-chains and excess of α-chains (2007) About Thalassemia, pp. 16-18. , Eleftheriou A, editor, Nicosia: The Thalassemia International Federation publisher
Schrier, S.L., Pathophysiology of thalassemia (2002) Curr Opin Hematol, 9, pp. 123-126
Bank, A., Regulation of human fetal hemoglobin: New players, new complexities (2006) Blood, 107, pp. 435-443
Voon, H.P., Wardan, H., Vadolas, J., Co-inheritance of alpha- and beta-thalassaemia in mice ameliorates thalassaemic phenotype (2007) Blood Cells Mol Dis, 39, pp. 184-188
Gambari, R., Fibach, E., Medicinal chemistry of fetal hemoglobin inducers for treatment of beta-thalassemia (2007) Curr Med Chem, 14, pp. 199-212
Xie, S.Y., Ren, Z.R., Zhang, J.Z., Guo, X.B., Wang, Q.X., Wang, S., Restoration of the balanced alpha/beta-globin gene expression in beta654-thalassemia mice using combined RNAi and antisense RNA approach (2007) Hum Mol Genet, 16, pp. 2616-2625
Bianchi, N., Borgatti, M., Gambari, R., Gene Silencing in thalassemia (2008) Minerva Biotecnologica, 20, pp. 51-55
Abes, S., Ivanova, G.D., Abes, R., Arzumanov, A.A., Williams, D., Owen, D., Peptide-based delivery of steric-block PNA oligonucleotides (2009) Methods Mol Biol, 480, pp. 85-99
Lundin, P., Johansson, H., Guterstam, P., Holm, T., Hansen, M., Langel, U., Distinct uptake routes of cell-penetrating peptide conjugates (2008) Bioconjug Chem, 19, pp. 2535-2542
Ivanova, G.D., Arzumanov, A., Abes, R., Yin, H., Wood, M.J., Lebleu, B., Improved cell-penetrating peptide-PNA conjugates for splicing redirection in HeLa cells and exon skipping in mdx mouse muscle (2008) Nucleic Acids Res, 36, pp. 6418-6428
Ivanova, G.D., Fabani, M.M., Arzumanov, A.A., Abes, R., Yin, H., Lebleu, B., PNA-peptide conjugates as intracellular gene control agents (2008) Nucleic Acids Symp Ser (Oxf), 52, pp. 31-32
Wright, D.G., Zhang, Y., Murphy, J.R., Effective delivery of antisense peptide nucleic acid oligomers into cells by anthrax protective antigen (2008) Biochem Biophys Res Commun, 376, pp. 200-205
Shiraishi, T., Hamzavi, R., Nielsen, P.E., Subnanomolar antisense activity of phosphonate-peptide nucleic acid (PNA) conjugates delivered by cationic lipids to HeLa cells (2008) Nucleic Acids Res, 36, pp. 4424-4432
Robertson, K.L., Yu, L., Armitage, B.A., Lopez, A.J., Peteanu, L.A., Fluorescent PNA probes as hybridization labels for biological RNA (2006) Biochemistry, 45, pp. 6066-6074
Rasmussen, F.W., Bendifallah, N., Zachar, V., Shiraishi, T., Fink, T., Ebbesen, P., Evaluation of transfection protocols for unmodified and modified peptide nucleic acid (PNA) oligomers (2006) Oligonucleotides, 16, pp. 43-57
Albertshofer, K., Siwkowski, A.M., Wancewicz, E.V., Esau, C.C., Watanabe, T., Nishihara, K.C., Structure-activity relationship study on a simple cationic peptide motif for cellular delivery of antisense peptide nucleic acid (2005) J Med Chem, 48, pp. 6741-6749
Karras, J.G., Maier, M.A., Lu, T., Watt, A., Manoharan, M., Peptide nucleic acids are potent modulators of endogenous pre-mRNA splicing of the murine interleukin-5 receptor-alpha chain (2001) Biochemistry, 40, pp. 7853-7859
Siwkowski, A.M., Malik, L., Esau, C.C., Maier, M.A., Wancewicz, E.V., Albertshofer, K., Identification and functional validation of PNAs that inhibit murine CD40 expression by redirection of splicing (2004) Nucleic Acids Res, 32, pp. 2695-2706
Wilusz, J.E., Devanney, S.C., Caputi, M., Chimeric peptide nucleic acid compounds modulate splicing of the bcl-x gene in vitro and in vivo (2005) Nucleic Acids Res, 33, pp. 6547-6554
El-Andaloussi, S., Johansson, H.J., Lundberg, P., Langel, U., Induction of splice correction by cell-penetrating peptide nucleic acids (2006) J Gene Med, 8, pp. 1262-1273
van Vliet, L., de Winter, C.L., van Deutekom, J.C., van Ommen, G.J., Aartsma-Rus, A., Assessment of the feasibility of exon 45-55 multiexon skipping for Duchenne muscular dystrophy (2008) BMC Med Genet, 9, pp. 105-111
Gao, Y., Zu, T., Low, W.C., Orr, H.T., McIvor, R.S., Antisense RNA sequences modulating the ataxin-1 message: Molecular model of gene therapy for spinocerebellar ataxia type 1, a dominant-acting unstable trinucleotide repeat disease (2008) Cell Transplant, 17, pp. 723-734
Davis, R.L., Homer, V.M., George, P.M., Brennan, S.O., A deep intronic mutation in FGB creates a consensus exonic splicing enhancer motif that results in afibrinogenemia caused by aberrant mRNA splicing, which can be corrected in vitro with antisense oligonucleotide treatment (2009) Hum Mutat, 30, pp. 221-227
Madsen, E.C., Morcos, P.A., Mendelsohn, B.A., Gitlin, J.D., In vivo correction of a Menkes disease model using antisense oligonucleotides (2008) Proc Natl Acad Sci USA, 105, pp. 3909-3914
Mascellani, N., Tagliavini, L., Gamberoni, G., Rossi, S., Marchesini, J., Taccioli, C., Using miRNA expression data for the study of human cancer (2008) Minerva Biotecnologica, 20, pp. 23-30
Habbe, N., Koorstra, J.B., Mendell, J.T., Offerhaus, G.J., Ryu, J.K., Feldmann, G., MicroRNA miR-155 is a biomarker of early pancreatic neoplasia (2009) Cancer Biol Ther, 8, pp. 25-31
Duisters, R.F., Tijsen, A.J., Schroen, B., Leenders, J.J., Lentink, V., van der Made, I., miR-133 and miR-30 regulate connective tissue growth factor: Implications for a role of microRNAs in myocardial matrix remodeling (2009) Circ Res, 104, pp. 170-178
Chen, J.F., Callis, T.E., Wang, D.Z., microRNAs and muscle disorders (2009) J Cell Sci, 122, pp. 13-20
Xiao, B., Guo, J., Miao, Y., Jiang, Z., Huan, R., Zhang, Y., Detection of miR-106a in gastric carcinoma and its clinical significance (2009) Clin Chim Acta, 400, pp. 97-102
Abes, S., Turner, J.J., Ivanova, G.D., Owen, D., Williams, D., Arzumanov, A., Efficient splicing correction by PNA conjugation to an R6-Penetratin delivery peptide (2007) Nucleic Acids Res, 35, pp. 4495-4502
Nielsen, P. E., Egholm, M., Berg, R. H., Buchardt, O., Sequence selective recognition of DNA by strand displacement with a thymine-substituted polyamide (1991) Science, 254, pp. 1497-150
Lundin, K. E., Good, L., Str mberg, R., Gr slund, A., Smith, C. I., Biological activity and biotechnological aspects of peptide nucleic acid (2006) Adv Genet, 56, pp. 1-51
Nielsen, P. E., Gene targeting using peptide nucleic acid (2005) Methods Mol Biol, 288, pp. 343-358
Pesce, C. D., Bolacchi, F., Bongiovanni, B., Cisotta, F., Capozzi, M., Diviacco, S., Anti-gene peptide nucleic acid targeted to proviral HIV-1 DNA inhibits in vitro HIV-1 replication (2005) Antiviral Res, 66, pp. 13-22
Bonham, M. A., Brown, S., Boyd, A. L., Brown, P. H., Bruckenstein, D. A., Hanvey, J. C., An assessment of the antisense properties of RNase H-competent and steric-blocking oligomers (1995) Nucleic Acids Res, 23, pp. 1197-1203
Weatherall, D. J., Clegg, J. B., (2001) The thalassemia syndromes, pp. 133-191. , Oxford: Blackwell Scientific
Ho, P. J., The regulation of beta globin gene expression and beta thalassemia (1999) Pathology, 31, pp. 315-324
Schrier, S. L., Pathophysiology of thalassemia (2002) Curr Opin Hematol, 9, pp. 123-126
Voon, H. P., Wardan, H., Vadolas, J., Co-inheritance of alpha- and beta-thalassaemia in mice ameliorates thalassaemic phenotype (2007) Blood Cells Mol Dis, 39, pp. 184-188
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PNA-mediated alteration of mRNA splicing
PNAs are expected to be very active in altering the splicing pattern of target RNA sequences. Several reports have been published on this specific task, focused on analysis of PNA altering splicing and vehiculated with a variety of delivery systems. The conclusions, here reviewed, are that PNAs altering splicing have important biological effects. In our own laboratory, we have studied the in vitro effects of a 14-mer PNA targeting a portion of the β-globin gene intron 1, suggesting that this strategy might be of interest to counteract the excess production of β-globin. Furthermore, the data obtained on the same 14-mer PNA, conjugated to the SV40 NLS peptide (β-globin-PNA/ NLS) to enhance the PNA concentration in cells, demonstrate that the NLS peptide does not interfere with inhibition of the splicing.
PNAs are expected to be very active in altering the splicing pattern of target RNA sequences. Several reports have been published on this specific task, focused on analysis of PNA altering splicing and vehiculated with a variety of delivery systems. The conclusions, here reviewed, are that PNAs altering splicing have important biological effects. In our own laboratory, we have studied the in vitro effects of a 14-mer PNA targeting a portion of the β-globin gene intron 1, suggesting that this strategy might be of interest to counteract the excess production of β-globin. Furthermore, the data obtained on the same 14-mer PNA, conjugated to the SV40 NLS peptide (β-globin-PNA/ NLS) to enhance the PNA concentration in cells, demonstrate that the NLS peptide does not interfere with inhibition of the splicing.
PNA-mediated alteration of mRNA splicing
| ▼ Biomolecular NMR: structural studies of proteins and peptides and drug discovery applications |
PNA-mediated alteration of mRNA splicing
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* From Prebiotic Chemistry to Supramolecular Biomedical Materials: Exploring the Properties of Self-Assembling Nucleobase-Containing Peptides (26 views)
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Impact Factor: 5.476
View Export to BibTeX Export to EndNote
Roviello GN
* Novel insights into nucleoamino acids: biomolecular recognition and aggregation studies of a thymine-conjugated L-phenyl alanine (366 views)
Amino Acids (ISSN: 1438-2199electronic, 0939-4451linking), 2018 May 15; DOI10.1007/s00726-01: 933-941.
Impact Factor: 3.173
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Impact Factor: 5.476
View Export to BibTeX Export to EndNote
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Impact Factor: 3.057
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Neve Polimeno M, Ierano C, D'Alterio C, Simona Losito N, Napolitano M, Portella L, Scognamiglio G, Tatangelo F, Maria Trotta A, Curley S, Costantini S, Liuzzi R, Izzo F, Scala S
* CXCR4 expression affects overall survival of HCC patients whereas CXCR7 expression does not (523 views)
Cell Mol Immunol (ISSN: 1672-7681), 2014 Nov 3; 12(4): 474-482.
Impact Factor: 4.112
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Zanoli LM, Licciardello M, D'Agata R, Lantano C, Calabretta A, Corradini R, Marchelli R, Spoto G
* Peptide nucleic acid molecular beacons for the detection of PCR amplicons in droplet-based microfluidic devices (266 views)
Anal Bioanal Chem (ISSN: 1618-2642), 2013 Jan; 405(2-3): 615-624.
Impact Factor: 3.578
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Roviello GN, Musumeci D, Bucci EM, Pedone C
* Synthesis of a diaminopropanoic acid-based nucleoamino acid and assembly of cationic nucleopeptides for biomedical applications (411 views)
Amino Acids (ISSN: 0939-4451, 1438-2199, 1438-2199electronic), 2012 Dec; 43(6): 2537-2543.
Impact Factor: 3.914
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Roviello GN, Roviello G, Musumeci D, Bucci EM, Pedone C
* Dakin-West reaction on 1-thyminyl acetic acid for the synthesis of 1, 3-bis(1-thyminyl)-2-propanone, a heteroaromatic compound with nucleopeptide-binding properties (398 views)
Amino Acids (ISSN: 1438-2199, 0939-4451, 1438-2199electronic), 2012 Oct; 43(4): 1615-1623.
Impact Factor: 3.914
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D'Agata R, Spoto G
* Artificial DNA and surface plasmon resonance (505 views)
Artificial Dna Pna & Xna (ISSN: 1949-095x), 2012 Apr; 3(2): N/D-N/D.
Impact Factor: 0
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Avitabile C, Saviano M, D'Andrea LD, Bianchi N, Fabbri E, Brognara E, Gambari R, Romanelli A
* Targeting pre-miRNA by peptide nucleic acids: a new strategy to interfere in the miRNA maturation (482 views)
Artif Dna Pna Xnaartificial Dna Pna & Xna (ISSN: 1949-9096, 1949-0968, 1949-095x), 2012 Apr; 3(2): 88-96.
Impact Factor: 0
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Zannetti A, Iommelli F, Speranza A, Salvatore M, Del Vecchio S
* 3'-deoxy-3'-18F-fluorothymidine (FLT) PET/CT to guide therapy with epidermal growth factor receptor (EGFR) antagonists and Bcl-xL inhibitors in non-small cell lung cancer (571 views)
J Nucl Med (ISSN: 0161-5505, 1535-5667, 1535-5667electronic), 2012 Mar 1; 53(3): 443-450.
Impact Factor: 5.774
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Zannetti A, Iommelli F, Speranza A, Salvatore M, Del Vecchio S
* 3'-deoxy-3'-18F-fluorothymidine PET/CT to guide therapy with epidermal growth factor receptor antagonists and Bcl-xL inhibitors in non-small cell lung cancer (479 views)
J Nucl Med (ISSN: 1535-5667, 0161-5505, 1535-5667electronic), 2012 Mar 1; 53(3): 443-450.
Impact Factor: 5.774
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Esposito G, De Falco F, Tinto N, Testa F, Vitagliano L, Tandurella IC, Iannone L, Rossi S, Rinaldi E, Simonelli F, Zagari A, Salvatore F
* Comprehensive mutation analysis (20 families) of the choroideremia gene reveals a missense variant that prevents the binding of REP1 with Rab geranylgeranyl transferase (450 views)
Hum Mutat (ISSN: 1059-7794, 1098-1004, 1098-1004electronic), 2011 Dec; 32(12): 1460-1469.
Impact Factor: 5.686
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Roviello GN, Musumeci D, Bucci EM, Pedone C
* Synthesis and characterization of a novel ester-based nucleoamino acid for the assembly of aromatic nucleopeptides for biomedical applications (477 views)
Int J Pharm (ISSN: 0378-5173, 1873-3476), 2011 Sep 30; 415(1-2): 206-210.
Impact Factor: 3.35
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Pensato S, Saviano M, Bianchi N, Borgatti M, Fabbri E, Gambari R, Romanelli A
* Gamma-Hydroxymethyl Pnas: Synthesis, Interaction With Dna And Inhibition Of Protein/Dna Interactions (350 views)
Bioorg Chem, 2010 Sep; 38(5): 196-201.
Impact Factor: 2.141
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Roviello GN, Crescenzo C, Capasso D, Di Gaetano S, Franco S, Bucci EM, Pedone C
* Synthesis of a novel Fmoc-protected nucleoaminoacid for the solid phase assembly of 4-piperidyl glycine/L-arginine-containing nucleopeptides and preliminary RNA interaction studies (431 views)
Amino Acids (ISSN: 0939-4451, 1438-2199, 1438-2199electronic), 2010 Sep; 39(3): 795-800.
Impact Factor: 4.106
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Del Vecchio S, Zannetti A, Iommelli F, Lettieri A, Brunetti A, Salvatore M
* Molecular imaging of tumor microenvironment (443 views)
Q J Nucl Med Mol Im (ISSN: 1824-4785, 1824-4478, 1824-4785linking), 2010 Jun; 54(3): 243-258.
Impact Factor: 1.724
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Del Vecchio S, Zannetti A, Iommelli F, Lettieri A, Brunetti A, Salvatore M
* Molecular imaging of tumor microenvironment: Challenges and perspectives (574 views)
Q J Nucl Med Mol Im (ISSN: 1824-4785, 1824-4478, 1824-4785linking), 2010 Jun; 54(3): 249-258.
Impact Factor: 1.724
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D'Agata R, Corradini R, Ferretti C, Zanoli L, Gatti M, Marchelli R, Spoto G
* Ultrasensitive detection of non-amplified genomic DNA by nanoparticle-enhanced surface plasmon resonance imaging (386 views)
Biosens Bioelectron (ISSN: 0956-5663, 1873-4235electronic), 2010 May 15; 25(9): 2095-2100.
Impact Factor: 5.361
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Roviello GN, Di Gaetano S, Capasso D, Cesarani A, Bucci EM, Pedone C
* Synthesis, spectroscopic studies and biological activity of a novel nucleopeptide with Moloney murine leukemia virus reverse transcriptase inhibitory activity (417 views)
Amino Acids (ISSN: 0939-4451, 1438-2199, 1438-2199electronic), 2010 May; 38(5): 1489-1496.
Impact Factor: 4.106
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Galasso G, De Rosa R, Piscione F, Iaccarino G, Vosa C, Sorriento D, Piccolo R, Rapacciuolo A, Walsh K, Chiariello M
* Myocardial expression of FOXO3a-Atrogin-1 pathway in human heart failure (245 views)
Eur J Heart Fail European Journal Of Heart Failure (ISSN: 1388-9842), 2010; 12(12): 1290-1296.
Impact Factor: 4.512
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Criscuolo C, Filla A, Coppola G, Rinaldi C, Carbone R, Pinto S, Wang Q, De Leva MF, Salvatore E, Banfi S, Brunetti A, Quarantelli M, Geschwind DH, Pappata S, De Michele G
* Two novel CYP7B1 mutations in Italian families with SPG5: A clinical and genetic study (331 views)
J Neurol (ISSN: 0340-5354, 1432-1459, 1432-1459electronic), 2009 Sep; 256(8): 1252-1257.
Impact Factor: 2.903
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Calabrese V, Cornelius C, Mancuso C, Pennisi G, Calafato S, Bellia F, Bates TE, Giuffrida Stella AM, Schapira T, Dinkova Kostova AT, Rizzarelli E
* Cellular stress response: a novel target for chemoprevention and nutritional neuroprotection in aging, neurodegenerative disorders and longevity (961 views)
Neurochemical Research (ISSN: 1573-6903, 0364-3190), 2008 Dec; 33(12): 2444-2471.
Impact Factor: 2.26
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Roviello GN, Musumeci D, Bucci EM, Castiglione M, Cesarani A, Pedone C, Piccialli G
* Evidences for complex formation between l-dabPNA and aegPNA (290 views)
Bioorg Med Chem Lett (ISSN: 0960-894x, 1464-3405, 1464-3405electronic), 2008 Sep 1; 18(17): 4757-4760.
Impact Factor: 2.531
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D'Agata R, Corradini R, Grasso G, Marchelli R, Spoto G
* Ultrasensitive detection of DNA by PNA and nanoparticle-enhanced surface plasmon resonance imaging (389 views)
Chembiochem (ISSN: 1439-4227, 1439-7633, 1439-7633electronic), 2008 Sep 1; 9(13): 2067-2070.
Impact Factor: 3.322
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Marchelli R, Saviano M, Licandro E, Spoto G, Borgatti M, Gambari R
* PNAs targeting the "RNA world": Micro RNA next? (382 views)
Minerva Biotecnol (ISSN: 1120-4826), 2008; 20(4): 183-188.
Impact Factor: 0.1
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Zanoli L, Dagata R, Spoto G
* Surface plasmon-based optical detection of DNA peptide nucleic acids (247 views)
Minerva Biotecnol (ISSN: 1120-4826), 2008; 20(4): 165-174.
Impact Factor: 0.1
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Moccia M, Musumeci D, Valente M, Roviello GN, Sapio R, Pedone C, Bucci EM
* Thermodynamic studies on PNA and PNA/DNA dendrimer formation (230 views)
Nucleosides Nucleotides Nucleic Acids (ISSN: 1525-7770), 2007; 26(8-9): 1173-1176.
Impact Factor: 0.723
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Saporito A, Marasco D, Chambery A, Botti P, Monti SM, Pedone C, Ruvo M
* The chemical synthesis of the GstI protein by NCL on a X-Met site (605 views)
Biopolymers (ISSN: 0006-3525, 0006-6352, 0006-3525print), 2006 Dec 5; 83(5): 508-518.
Impact Factor: 2.48
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Roviello GN, Moccia M, Sapio R, Valente M, Bucci EM, Castiglione M, Pedone C, Perretta G, Benedetti E, Musumeci D
* Synthesis, Characterization And Hybridization Studies Of New Nucleo-Gamma-Peptides Based On Diaminobutyric Acid (466 views)
J Pept Sci (ISSN: 1075-2617, 1099-1387, 1075-2617print), 2006 Dec; 12(12): 829-835.
Impact Factor: 1.801
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Cittadini A, Monti MG, Iaccarino G, Di Rella F, Tsichlis PN, Di Gianni A, Strömer H, Sorriento D, Peschle C, Trimarco B, Saccà L, Condorelli G
* Adenoviral gene transfer of Akt enhances myocardial contractility and intracellular calcium handling (526 views)
Gene Ther (ISSN: 0969-7128, 0969-7128linking), 2006; 13(1): 8-19.
Impact Factor: 4.782
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Borgatti M, Boyd DD, Lampronti I, Bianchi N, Fabbri E, Saviano M, Romanelli A, Pedone C, Gambari R
* Decoy molecules based on PNA-DNA chimeras and targeting Sp1 transcription factors inhibit the activity of urokinase-type plasminogen activator receptor (uPAR) promoter (379 views)
Oncology Research (ISSN: 0965-0407), 2005; 15(7-8): 373-383.
Impact Factor: 1.844
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Borgatti M, Finotti A, Romanelli A, Saviano M, Bianchi N, Lampronti I, Lambertini E, Penolazzi L, Nastruzzi C, Mischiati C, Piva R, Pedone C, Gambari R
* Peptide nucleic acids (PNA)-DNA chimeras targeting transcription factors as a tool to modify gene expression (468 views)
Curr Drug Targets (ISSN: 1389-4501), 2004 Nov; 5(8): 735-744.
Impact Factor: 4.104
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Agmon I, Amit M, Auerbach T, Bashan A, Baram D, Bartels H, Berisio R, Greenberg I, Harms J, Hansen HAS, Kessler M, Pyetan E, Schluenzen F, Sittner A, Yonath A, Zarivach R
* Ribosomal crystallography: A flexible nucleotide anchoring tRNA translocation, facilitates peptide-bond formation, chirality discrimination and antibiotics synergism (292 views)
Febs Lett (ISSN: 0014-5793, 0014-5793print, 1873-3468electronic), 2004 Jun 1; 567(1): 20-26.
Impact Factor: 3.843
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Cortesi R, Mischiati C, Borgatti M, Breda L, Romanelli A, Saviano M, Pedone C, Gambari R, Nastruzzi C
* Formulations for natural and peptide nucleic acids based on cationic polymeric submicron particles (370 views)
The Aaps Journal (ISSN: 1522-1059, 1550-7416), 2004 Jan 20; 6(1): N/D-N/D.
Impact Factor: 1.938
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Musumeci D, Roviello GN, Valente M, Sapio R, Pedone C, Bucci EM
* New synthesis of PNA-3′DNA linker monomers, useful building blocks to obtain PNA/DNA chimeras (399 views)
Biopolymers, 2004; 76(6): 535-542.
Impact Factor: 2.248
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Gambari R, Borgatti M, Romanelli A, Capasso D, Saviano M, Bianchi N, Lampronti I, Mischiati C, Pedone C
* Transcription factor decoy (TFD) activity of peptide nucleic acids (PNA)-DNA chimeras (226 views)
International Journal Of Molecular Medicine (ISSN: 1107-3756), 2003; 12(Supplement1): N/D-N/D.
Impact Factor: 1.94
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Romanelli A, Pedone C, Saviano M, Bianchi N, Borgatti M, Mischiati C, Gambari R
* Molecular interactions between nuclear factor κB (NF-κB) transcription factors and a PNA-DNA chimera mimicking NF-κB binding sites (403 views)
Eur J Biochem (ISSN: 0014-2956, 0014-2956print, 0014-2956linking), 2001 Dec; 268(23): 6066-6075.
Impact Factor: 2.849
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Romanelli A, Pedone C, Saviano M, Bianchi N, Borgatti M, Mischiati C, Gambari R
* Molecular interactions with nuclear factor kappaB (NF-kappaB) transcription factors of a PNA-DNA chimera mimicking NF-kappaB binding sites (408 views)
Eur J Biochem (ISSN: 0014-2956print, 0014-2956linking), 2001 Dec; 268(23): 6066-6075.
Impact Factor: 4.779
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Pasquali D, Bellastella A, Valente A, Botti G, Capasso I, del Vecchio S, Salvatore M, Colantuoni V, Sinisi AA
Retinoic acid receptors α, β and γ, and cellular retinol binding protein-I expression in breast fibrocystic disease and cancer (283 views)
Eur J Endocrinol (ISSN: 0804-4643), 1997 Oct; 137(4): 410-414.
Impact Factor: 1.968
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FRITTITTA L, VIGNERI R, PAPA V, GOLDFINE I, GRASSO G, TRISCHITTA V
Structural and functional studies of insulin receptors in human breast cancer (420 views)
Breast Cancer Res Tr Kluwer Academic Publishers (ISSN: 0167-6806), 1993; 25(1): 73-82.
Impact Factor: 2.43
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Papa V, Reese CC, Brunetti A, Vigneri R, Siiteri PK, Goldfine ID
Progestins increase insulin receptor content and insulin stimulation of growth in human breast carcinoma cells (434 views)
Cancer Res (ISSN: 0008-5472, 1538-7445, 1538-7445electronic), 1990 Dec 15; 50(24): 7858-7862.
Impact Factor: 8.426
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46 Records (43 excluding Abstracts).
Total impact factor: 149.704 (145.826 excluding Abstracts).
Total 5 year impact factor: 154.116 (147.244 excluding Abstracts).
Total impact factor: 149.704 (145.826 excluding Abstracts).
Total 5 year impact factor: 154.116 (147.244 excluding Abstracts).
316 views. Last view on Wednesday 20 July 2022, 11:36:41
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