Cerebellar Astrocyte Transduction as Gene Therapy for Megalencephalic Leukoencephalopathy-Reference-Cited by-同舟云学术

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Cerebellar Astrocyte Transduction as Gene Therapy for Megalencephalic Leukoencephalopathy

Published:2020-05-05 Issue:4 Volume:17 Page:2041-2053
ISSN:1933-7213
Container-title:Neurotherapeutics
language:en
Short-container-title:Neurotherapeutics

Author:

Sánchez Angela,García-Lareu Belén,Puig Meritxell,Prat Esther,Ruberte Jesús,Chillón Miguel,Nunes Virginia,Estévez Raul,Bosch Assumpció

Funder

AGAUR

European Leukodystrophy Association

Ministerio de Ciencia e Innovación

Hugo Foundation

Instituto de Salud Carlos III

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),Clinical Neurology,Pharmacology

Link

https://link.springer.com/content/pdf/10.1007/s13311-020-00865-y.pdf

Reference47 articles.

1. van der Knaap MS, Barth PG, Stroink H et al. Leukoencephalopathy with swelling and a discrepantly mild clinical course in eight children. Ann Neurol 1995; 37:324-334.

2. Singhal BS, Gursahani RD, Udani VP et al. Megalencephalic leukodystrophy in an Asian Indian ethnic group. Pediatr Neurol 1996; 14:291-296.

3. Ben-Zeev B, Gross V, Kushnir T et al. Vacuolating megalencephalic leukoencephalopathy in 12 Israeli patients. J Child Neurol 2001; 16:93-99.

4. Harbord MG, Harden A, Harding B et al. Megalencephaly with dysmyelination, spasticity, ataxia, seizures and distinctive neurophysiological findings in two siblings. Neuropediatrics 1990; 21:164-168.

5. Leegwater PAJ, Yuan BQ, van der Steen J et al. Mutations of MLC1 (KIAA0027), encoding a putative membrane protein, cause megalencephalic leukoencephalopathy with subcortical cysts. Am J Hum Genet 2001; 68:831-838.

Cited by 8 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Megalencephalic leukoencephalopathy with subcortical cysts: a variant update and review of the literature;Frontiers in Genetics;2024-02-29

2. Gene and Cellular Therapies for Leukodystrophies;Pharmaceutics;2023-10-24

3. Characterization of an MLC patient carrying two MLC1 variants showing radiological improvement;Brain Disorders;2023-09

4. Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies;Frontiers in Cellular Neuroscience;2021-06-22

5. Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair;Frontiers in Cellular Neuroscience;2021-04-23

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