Gene Therapies in Motor Neuron Diseases ALS and SMA

被引:0
作者
Guenther, Rene [1 ]
机构
[1] Tech Univ Dresden, Univ Hosp Carl Gustav Carus, Klin & Poliklin Neurol, Fetscherstr 74, D-01307 Dresden, Germany
关键词
gene therapy; motor neuron diseases; ALS; SMA; SPINAL MUSCULAR-ATROPHY; AMYOTROPHIC-LATERAL-SCLEROSIS; ANTISENSE OLIGONUCLEOTIDE TOFERSEN; FLAIL ARM SYNDROME; NATURAL-HISTORY; MOLECULAR ANALYSIS; SHAM CONTROL; NUSINERSEN; PHENOTYPE; RISDIPLAM;
D O I
10.1055/a-2002-5215
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
In the past, the diagnosis of motor neuron diseases such as amyotrophic lateral sclerosis (ALS) and 5q-associated spinal muscular atrophy (SMA) meant powerlessness in the face of seemingly untreatable diseases with severe motor-functional limitations and sometimes fatal courses. Recent advances in an understanding of the genetic causalities of these diseases, combined with success in the development of targeted gene therapy strategies, spell hope for effective, innovative therapeutic approaches, pioneering the ability to treat neurodegenerative diseases. While gene therapies have been approved for SMA since a few years, gene therapy research in ALS is still in clinical trials with encouraging results. This article provides an overview of the genetic background of ALS and SMA known to date and gene therapy approaches to them with a focus on therapy candidates that are in clinical trials or have already gained market approval.
引用
收藏
页码:153 / 163
页数:11
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