Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia-Reference-Cited by-同舟云学术

Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia

Published:2023-09 Issue: Volume:30 Page:181-190
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Chandler Randy J.,Di Pasquale Giovanni,Choi Eun-Young,Chang David,Smith Stephanie N.,Sloan Jennifer L.,Hoffmann Victoria,Li Lina,Chiorini John A.,Venditti Charles P.^ORCID

Funder

National Human Genome Research Institute

National Institute of Dental and Craniofacial Research

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference46 articles.

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3. Localisation of enzymic defect in propionicacidaemia;Gompertz;Lancet,1970

4. Inherited propionyl-Coa carboxylase deficiency in "ketotic hyperglycinemia";Hsia;J. Clin. Invest.,1971

5. Crystal structure of the alpha(6)beta(6) holoenzyme of propionyl-coenzyme A carboxylase;Huang;Nature,2010

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