Location matters for brain gene therapy in lysosomal disorders

IF 50 1区医学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY

Nature Medicine Pub Date : 2025-08-19 DOI:10.1038/s41591-025-03914-1

Guilherme Baldo

引用次数: 0

Abstract

A study demonstrates the feasibility of a multi-site infusion approach to ensure broad distribution of an adeno-associated virus-based gene therapy for GM2 gangliosidosis, providing valuable lessons for the treatment of this and other rare lysosomal storage disorders.

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溶酶体疾病脑基因治疗的位置问题。

一项研究证明了多位点输注方法的可行性，以确保腺相关病毒为基础的GM2神经节脂质病基因治疗的广泛分布，为治疗这种疾病和其他罕见的溶酶体储存疾病提供了宝贵的经验。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Nature Medicine 医学-生化与分子生物学

CiteScore

100.90

自引率

0.70%

发文量

525

审稿时长

1 months

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