1028. Use of Antisense Oligonucleotides To Restore Activity in Mutant p53
نویسندگان
چکیده
منابع مشابه
Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy
Dominant-negative mutations in the genes that encode the three major α chains of collagen type VI, COL6A1, COL6A2, and COL6A3, account for more than 50% of Ullrich congenital muscular dystrophy patients and nearly all Bethlem myopathy patients. Gapmer antisense oligonucleotides (AONs) are usually used for gene silencing by stimulating RNA cleavage through the recruitment of an endogenous endonu...
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Olubunmi Afonja, Takashi Shimamoto, John E. Smith, Jr., Long Cui, and Kenichi Takeshita Hematology Division, Department of Medicine, Kaplan Cancer Center and Department of Pediatrics, New York University Medical Center, New York, NY. First Department of Internal Medicine, Tokyo Medical College, Tokyo, Japan and Hematology-Oncology Division, Metropolitan Hospital, New York Medical College, New Y...
متن کاملAntisense Oligonucleotides: problems with use and solutions
Les oligonucléotides antisens (OAS) montrent un grand potentiel comme outil moléculaire et comme nouvel agent thérapeutique. Cependant, plusieurs problèmes limitent leur utilisation, en particulier leur toxicité, leurs effets indésirables et leur faible pénétration dans les cellules. Chez l'homme, les études cliniques concernant l'utilisation des oligonucléotides phosphorotioates (PS-ODNs) ont ...
متن کاملBest minimally modified antisense oligonucleotides according to cell nuclease activity.
Minimally modified oligonucleotides belong to the second-generation antisense class. They are phosphodiester oligonucleotides with a minimum of phosphorothioate linkages in order to be protected against serum and cellular exonucleases and endonucleases. They activate RNase H, have weak interactions with proteins, and have thus a better antisense efficiency. Two of them have been designed from a...
متن کاملA novel dysferlin mutant pseudoexon bypassed with antisense oligonucleotides
OBJECTIVE Mutations in dysferlin (DYSF), a Ca(2+)-sensitive ferlin family protein important for membrane repair, vesicle trafficking, and T-tubule function, cause Miyoshi myopathy, limb-girdle muscular dystrophy type 2B, and distal myopathy. More than 330 pathogenic DYSF mutations have been identified within exons or near exon-intron junctions. In ~17% of patients who lack normal DYSF, only a s...
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ژورنال
عنوان ژورنال: Molecular Therapy
سال: 2006
ISSN: 1525-0016
DOI: 10.1016/j.ymthe.2006.08.1123