Preclinical Evaluation of a Clinical Candidate AAV8 Vector for Ornithine Transcarbamylase (OTC) Deficiency Reveals Functional Enzyme from Each Persisting Vector Genome

Ornithine transcarbamylase deficiency (OTCD), the most common and severe urea cycle disorder, is an excellent model for developing liver-directed gene therapy. No curative therapy exists except for liver transplantation which is limited by available donors and carries significant risk of mortality a...

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Autores principales: Wang, Lili, Morizono, Hiroki, Lin, Jianping, Bell, Peter, Jones, David, McMenamin, Deirdre, Yu, Hongwei, Batshaw, Mark L., Wilson, James M.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2011
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Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3270700/
https://ncbi.nlm.nih.gov/pubmed/22133298
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgme.2011.10.020
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