Article

Preclinical Evaluation of AAV9-coSMN1 Gene Therapy for Spinal Muscular Atrophy：Efficacy and Safety in Mouse Models and Non-Human Primates

2025-03-31

Abstract excerpt

<title>Abstract</title> <p><bold>Background</bold> Spinal muscular atrophy (SMA) is a severe neuromuscular disorder caused by the loss of motor neurons in the spinal cord. Our team has initiated clinical trials using adeno-associated virus serotype 9 (AAV9) vectors carrying a codon-optimized SMN1 (coSMN1) gene, delivered via intrathecal (IT) injection. Here, we present the preclinical research that laid the groun...

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Literature Corpus work: 274a959a-df15-5a70-b6fd-a9e3bad16f3a
DOI: 10.21203/rs.3.rs-5051200/v1

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