Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia

Isolated methylmalonic acidemia (MMA), a group of autosomal recessive inborn errors of metabolism, is most commonly caused by complete (mut(0)) or partial (mut(−)) deficiency of the enzyme methylmalonyl-CoA mutase (MUT). The severe metabolic instability and increased mortality experienced by many af...

Deskribapen osoa

Gorde:
Xehetasun bibliografikoak
Argitaratua izan da:Hum Gene Ther
Egile Nagusiak: Harrington, Elizabeth A., Sloan, Jennifer L., Manoli, Irini, Chandler, Randy J., Schneider, Mark, McGuire, Peter J., Calcedo, Roberto, Wilson, James M., Venditti, Charles P.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: Mary Ann Liebert, Inc. 2016
Gaiak:
Research Articles
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC4841085/
https://ncbi.nlm.nih.gov/pubmed/26790480
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.092
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