Introduction to Gene Therapy

被引：0
作者：
Gasser, Thomas [1 ,2 ]
机构：
[1] Hertie Inst Klin Hirnforsch, Zentrum Neurol, Abt Neurodegenerat Erkrankungen, Tubingen, Germany
[2] Hertie Inst Klin Hirnforschung, Zent Neurol, Abt Neurodegenerat Erkrankungen, Hoppe Seyler Str 3, D-72076 Tubingen, Germany
来源：
FORTSCHRITTE DER NEUROLOGIE PSYCHIATRIE | 2023年 / 91卷 / 04期
关键词：
gene therapy; viral vectors; antisense oligonucleotides; siRNA; ANTISENSE OLIGONUCLEOTIDE; REPLACEMENT THERAPY;
D O I：
10.1055/a-2002-5176
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
After many years of research and numerous setbacks, there are now undeniable success stories of gene therapies, namely the direct modification of genetic information on the DNA or RNA level. Both "ex vivo " strategies, i.e. the genetic manipulation of patient cells in a dish, as well as "in vivo " approaches are being used successfully. In addition to the supplementation of defective genes, the use of the CRISPR-Cas9 system to alter nuclear DNA sequences and the sequence-specific interference with the transcriptional process on the RNA level can be designated as gene therapies in a broad sense.
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收藏
页码：129 / 134
页数：6
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