A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice-Reference-Cited by-同舟云学术

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A blood-brain barrier-penetrant AAV gene therapy improves neurological function in symptomatic mucolipidosis IV mice

Published:2024-06 Issue:2 Volume:32 Page:101269
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Sangster Madison L.,Bishop Martha M.,Yao Yizheng,Feitor Jessica F.,Shahriar Sanjid,Miller Maxwell E.,Chekuri Anil K.,Budnik Bogdan,Bei Fengfeng,Grishchuk Yulia^ORCID

Funder

Mass General Brigham Inc

Publisher

Elsevier BV

Reference45 articles.

1. A physical and transcript map of the MCOLN1 gene region on human chromosome 19p13.3-p13.2;Acierno;Genomics,2001

2. Congenital corneal clouding with abnormal systemic storage bodies: a new variant of mucolipidosis;Berman;J. Pediatr.,1974

3. Mucolipidosis type IV: the origin of the disease in the Ashkenazi Jewish population;Raas-Rothschild;Eur. J. Hum. Genet.,1999

4. Mapping of the mucolipidosis type IV gene to chromosome 19p and definition of founder haplotypes;Slaugenhaupt;Am. J. Hum. Genet.,1999

5. Identification of the gene causing mucolipidosis type IV;Bargal;Nat. Genet.,2000

Cited by 2 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. Plasma Proteomic Signature of Mucolipidosis Type IV;2024-07-29

2. Gene therapy for CNS disorders: modalities, delivery and translational challenges;Nature Reviews Neuroscience;2024-06-19

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