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

Mucolipidosis IV (MLIV) is a rare, autosomal recessive, lysosomal disease characterized by intellectual disability, motor deficits, and progressive vision loss. Using adeno-associated vector 9 (AAV9) and AAV-PHP.B as delivery vectors, we previously demonstrated the feasibility of modifying disease c...

Whakaahuatanga katoa

I tiakina i:
Ngā kaituhi matua: Madison L. Sangster, Martha M. Bishop, Yizheng Yao, Jessica F. Feitor, Sanjid Shahriar, Maxwell E. Miller, Anil K. Chekuri, Bogdan Budnik, Fengfeng Bei, Yulia Grishchuk
Hōputu: Artigo
Reo:Inglês
I whakaputaina: Elsevier 2024-06-01
Rangatū:Molecular Therapy: Methods & Clinical Development
Ngā marau:
TRPML1
lysosomal disorder
AAV
adeno-associated vector
gene transfer
pediatric neurological disease
Urunga tuihono:http://www.sciencedirect.com/science/article/pii/S2329050124000858
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