Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia.

Chandler, Randy J; Venditti, Charles P

Chandler, Randy J; Venditti, Charles P.

Affiliation

Chandler RJ; National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.
Venditti CP; National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.

J Inherit Metab Dis ; 47(1): 63-79, 2024 Jan.

Article in En | MEDLINE | ID: mdl-37530705

Subject(s)

Amino Acid Metabolism, Inborn Errors; Liver Transplantation; Propionic Acidemia; Animals; Mice; Humans; Propionic Acidemia/genetics; Propionic Acidemia/therapy; Propionic Acidemia/complications; Amino Acid Metabolism, Inborn Errors/genetics; Amino Acid Metabolism, Inborn Errors/therapy; Amino Acid Metabolism, Inborn Errors/complications; Liver Transplantation/adverse effects; Genetic Therapy; Disease Models, Animal; Methylmalonic Acid

Key words

AAV; CRISPR; MMA; PA; adeno-associated virus; gene therapy; genome editing; homologous recombination; mRNA therapy; methylmalonic acidemia; mouse models; organic acidemia; propionic acidemia

Fulltext

XML

PubMed Links

Search on Google

Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Liver Transplantation / Propionic Acidemia / Amino Acid Metabolism, Inborn Errors Limits: Animals / Humans Language: En Journal: J Inherit Metab Dis Year: 2024 Type: Article Affiliation country: United States

Fulltext

XML

PubMed Links

Search on Google