Academic Journal

Advances in gene therapy for neurogenetic diseases: a brief review.

التفاصيل البيبلوغرافية
العنوان: Advances in gene therapy for neurogenetic diseases: a brief review.
المؤلفون: Xie, Ying-Xuan, Lv, Wen-Qi, Chen, Yi-Kun, Hong, Shunyan, Yao, Xiang-Ping, Chen, Wan-Jin, Zhao, Miao
المصدر: Journal of Molecular Medicine; Mar2022, Vol. 100 Issue 3, p385-394, 10p
مصطلحات موضوعية: GENE therapy, GENETIC vectors, GENETIC transformation, GENOME editing, TREATMENT effectiveness, THERAPEUTICS, NEUROLOGICAL disorders, RNA editing
مستخلص: Neurogenetic diseases are neurological conditions with a genetic cause (s). There are thousands of neurogenetic diseases, and most of them are incurable. The development of bioinformatics and elucidation of the mechanism of pathogenesis have allowed the development of gene therapy approaches, which show great potential in treating neurogenetic diseases. Viral vectors delivery, antisense oligonucleotides, gene editing, RNA interference, and burgeoning viroid delivery technique are promising gene therapy strategies, and commendable therapeutic effects in the treatment of neurogenetic diseases have been achieved (Fig. 1). This review highlights a sampling of advances in gene therapies for neurogenetic disorders. [ABSTRACT FROM AUTHOR]
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قاعدة البيانات: Complementary Index
الوصف
تدمد:09462716
DOI:10.1007/s00109-021-02167-y