杜兴氏肌肉萎缩症的 AAV 基因疗法：从 3 期试验中汲取的经验教训。

IF 4.6 3区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY Gene Therapy Pub Date : 2024-10-23 DOI:10.1038/s41434-024-00494-6

Giovanni Baranello, Francesco Muntoni

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AAV gene therapy for Duchenne Muscular Dystrophy: lessons learned from a phase 3 trial

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来源期刊

Gene Therapy 医学-生化与分子生物学

CiteScore

9.70

自引率

2.00%

发文量

审稿时长

4-8 weeks

期刊介绍： Gene Therapy covers both the research and clinical applications of novel therapeutic techniques based on a genetic component. Over the last few decades, significant advances in technologies ranging from identifying novel genetic targets that cause disease through to clinical studies, which show therapeutic benefit, have elevated this multidisciplinary field to the forefront of modern medicine.

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