Phase I clinical trial of intracerebral injection of lentiviral-ABCD1 for the treatment of cerebral adrenoleukodystrophy-Reference-Cited by-同舟云学术

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Phase I clinical trial of intracerebral injection of lentiviral-ABCD1 for the treatment of cerebral adrenoleukodystrophy

Published:2024-08 Issue:16 Volume:69 Page:2596-2603
ISSN:2095-9273
Container-title:Science Bulletin
language:en
Short-container-title:Science Bulletin

Author:

Wang Qiu-Hong,Wang Jing,Ling Zhi-Pei,Cui Zhi-Qiang,Gong Jie,Zhang Rui,Li Shi-Jun,Wang Yang-Yang,Yang Rui,Huang De-Hui,He Wen,Gao Jing,Feng Chen,Hu Pei-Li,Liu Li-Ying,Chang Lung-Ji,Zou Li-Ping

Publisher

Elsevier BV

Reference34 articles.

1. Adrenoleukodystrophy: Phenotype, genetics, pathogenesis and therapy;Moser;Brain,1997

2. Update on clinical ex vivo hematopoietic stem cell gene therapy for inherited monogenic diseases;Tucci;Mol Ther,2021

3. Microglia damage precedes major myelin breakdown in X-linked adrenoleukodystrophy and metachromatic leukodystrophy;Bergner;Glia,2019

4. Oxidative stress, mitochondrial and proteostasis malfunction in adrenoleukodystrophy: A paradigm for axonal degeneration;Fourcade;Free Radic Bio Med,2015

5. X-linked adrenoleukodystrophy (X-ALD): Clinical presentation and guidelines for diagnosis, follow-up and management;Engelen;Orphanet J Rare Dis,2012

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