Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs
暂无分享,去创建一个
D. Corey | I. Bezprozvanny | Masayuki Matsui | Jun Wu | K. Arar | Keith T. Gagnon | K. Gagnon | J. Schwartz | Jiaxin Hu | Sylvie Gabillet
[1] R. Friedlander,et al. Allele‐specific silencing of mutant Huntington’s disease gene , 2009, Journal of neurochemistry.
[2] D. Sah,et al. Novel Therapeutic Modalities to Address Nondrugable Protein Interaction Targets , 2009, Neuropsychopharmacology.
[3] N. Déglon,et al. Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease , 2008, PloS one.
[4] E. Burright,et al. Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts. , 2008, Human gene therapy.
[5] S. Kauppinen,et al. Chapter 5:Locked Nucleic Acid: Properties and Therapeutic Aspects , 2008 .
[6] K. G. Rajeev,et al. Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits , 2007, Proceedings of the National Academy of Sciences.
[7] Ralph Weissleder,et al. MicroRNA-21 knockdown disrupts glioma growth in vivo and displays synergistic cytotoxicity with neural precursor cell delivered S-TRAIL in human gliomas. , 2007, Cancer research.
[8] H. Zoghbi,et al. Trinucleotide repeat disorders. , 2007, Annual review of neuroscience.
[9] D. Corey,et al. Inhibiting gene expression with peptide nucleic acid (PNA)--peptide conjugates that target chromosomal DNA. , 2007, Biochemistry.
[10] H. Paulson,et al. Dominantly Inherited Ataxias: Lessons Learned from Machado-Joseph Disease/Spinocerebellar Ataxia Type 3 , 2007, Seminars in neurology.
[11] David R Corey,et al. RNA learns from antisense. , 2007, Nature chemical biology.
[12] M. Manoharan,et al. RNAi therapeutics: a potential new class of pharmaceutical drugs , 2006, Nature chemical biology.
[13] M. MacDonald,et al. Huntington's disease: seeing the pathogenic process through a genetic lens. , 2006, Trends in biochemical sciences.
[14] B. Monia,et al. Antisense oligonucleotide therapy for neurodegenerative disease. , 2006, The Journal of clinical investigation.
[15] J. Schelter,et al. Designing siRNA That Distinguish between Genes That Differ by a Single Nucleotide , 2006, PLoS genetics.
[16] H. Paulson,et al. Allele-specific RNA interference for neurological disease , 2006, Gene Therapy.
[17] B. Davidson,et al. RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases , 2006, Gene Therapy.
[18] I. Kanazawa,et al. Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA , 2005, Neuroscience Research.
[19] D. Corey,et al. Inhibiting transcription of chromosomal DNA with antigene peptide nucleic acids , 2005, Nature chemical biology.
[20] B. Armitage,et al. RNA guanine quadruplex invasion by complementary and homologous PNA probes. , 2005, Journal of the American Chemical Society.
[21] H. Paulson,et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. , 2005, Proceedings of the National Academy of Sciences of the United States of America.
[22] R. Llinás,et al. Disturbed Ca2+ signaling and apoptosis of medium spiny neurons in Huntington's disease. , 2005, Proceedings of the National Academy of Sciences of the United States of America.
[23] Jacek Krol,et al. RNA structure of trinucleotide repeats associated with human neurological diseases. , 2003, Nucleic acids research.
[24] E. Simpson,et al. Selective striatal neuronal loss in a YAC128 mouse model of Huntington disease. , 2003, Human molecular genetics.
[25] Haibin Xia,et al. Allele-specific silencing of dominant disease genes , 2003, Proceedings of the National Academy of Sciences of the United States of America.
[26] V. Erdmann,et al. Design of antisense oligonucleotides stabilized by locked nucleic acids. , 2002, Nucleic acids research.
[27] L. Thompson,et al. Antisense-mediated down-regulation of the human huntingtin gene. , 2000, The Journal of pharmacology and experimental therapeutics.
[28] T. Hökfelt,et al. Potent and nontoxic antisense oligonucleotides containing locked nucleic acids. , 2000, Proceedings of the National Academy of Sciences of the United States of America.
[29] Jacqueline K. White,et al. Huntingtin is required for neurogenesis and is not impaired by the Huntington's disease CAG expansion , 1997, Nature Genetics.
[30] P. Nielsen,et al. Antisense properties of duplex- and triplex-forming PNAs. , 1996, Nucleic acids research.
[31] D. Corey. 48000-FOLD ACCELERATION OF HYBRIDIZATION BY CHEMICALLY MODIFIED OLIGONUCLEOTIDES , 1995 .
[32] S. Floresco,et al. Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes , 1995, Cell.
[33] G. Sobue,et al. Molecular features of the CAG repeats and clinical manifestation of Machado-Joseph disease. , 1995, Human molecular genetics.
[34] Paul W Goldberg,et al. A worldwide study of the Huntington's disease mutation. The sensitivity and specificity of measuring CAG repeats. , 1994, The New England journal of medicine.
[35] Joseph B. Martin. Huntington's disease , 1984, Neurology.