首页>
外国专利>
Single stranded oligonucleotides complementary to repetitive elements for treating dna repeat instability associated genetic disorders
Single stranded oligonucleotides complementary to repetitive elements for treating dna repeat instability associated genetic disorders
展开▼
机译:与重复元件互补的单链寡核苷酸,用于治疗dna重复不稳定性相关的遗传疾病
展开▼
页面导航
摘要
著录项
相似文献
摘要
The current invention provides for methods and medicaments that apply oligonucleotide molecules complementary only to a repetitive sequence in a human gene transcript, for the manufacture of a medicament for the diagnosis, treatment or prevention of a cis-element repeat instability associated genetic disorders in humans. The invention hence provides a method of treatment for cis-element repeat instability associated genetic disorders. The invention also pertains to modified oligonucleotides which can be applied in method of the invention to prevent the accumulation and/or translation of repeat expanded transcripts in cells.
展开▼