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...

Celý popis

Uloženo v:
Podrobná bibliografie
Hlavní autoři: Wang, Lili, Morizono, Hiroki, Lin, Jianping, Bell, Peter, Jones, David, McMenamin, Deirdre, Yu, Hongwei, Batshaw, Mark L., Wilson, James M.
Médium: Artigo
Jazyk:Inglês
Vydáno: 2011
Témata:
Article
On-line přístup: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
Tagy: Přidat tag
Žádné tagy, Buďte první, kdo otaguje tento záznam!

Podobné jednotky