Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9-Reference-Cited by-同舟云学术

Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9

Published:2022-12 Issue: Volume:27 Page:61-72
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,Sloan Jennifer L.,McCoy Samantha,Hubbard Brandon T.,Kilts Tina M.,Manoli Irini,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

Reference51 articles.

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3. The natural history of the inherited methylmalonic acidemias;Matsui;N. Engl. J. Med.,1983

4. Long-term outcome in methylmalonic acidurias is influenced by the underlying defect (mut0, mut-cblA, cblB);Hörster;Pediatr. Res.,2007

5. Methylmalonic aciduria. An inborn error of metabolism leading to chronic metabolic acidosis;Oberholzer;Arch. Dis. Child.,1967

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