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TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND DISORDERS ASSOCIATED WITH THE SPINAL CORD

机译:脊髓性肌萎缩性侧索硬化症和疾病的治疗

摘要

The present disclosure relates to AAVs encoding a SOD1 targeting polynucleotide which may be used to treat amyotrophic lateral sclerosis (ALS) and delivery methods for the treatment of spinal cord related disorders including ALS.
机译:本公开涉及编码可用于治疗肌萎缩性侧索硬化(ALS)的靶向SOD1多核苷酸的AAV以及用于治疗包括ALS在内的脊髓相关疾病的递送方法。

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