溶酶体疾病脑基因治疗的位置问题。

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

摘要

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。

Location matters for brain gene therapy in lysosomal disorders

查看原文本刊更多论文

Location matters for brain gene therapy in lysosomal disorders

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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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

期刊介绍： Nature Medicine is a monthly journal publishing original peer-reviewed research in all areas of medicine. The publication focuses on originality, timeliness, interdisciplinary interest, and the impact on improving human health. In addition to research articles, Nature Medicine also publishes commissioned content such as News, Reviews, and Perspectives. This content aims to provide context for the latest advances in translational and clinical research, reaching a wide audience of M.D. and Ph.D. readers. All editorial decisions for the journal are made by a team of full-time professional editors. Nature Medicine consider all types of clinical research, including: -Case-reports and small case series -Clinical trials, whether phase 1, 2, 3 or 4 -Observational studies -Meta-analyses -Biomarker studies -Public and global health studies Nature Medicine is also committed to facilitating communication between translational and clinical researchers. As such, we consider “hybrid” studies with preclinical and translational findings reported alongside data from clinical studies.