Long-term correction of ornithine transcarbamylase deficiency in Spf-Ash mice with a translationally optimized AAV vector

Ornithine transcarbamylase deficiency (OTCD) is an X-linked liver disorder caused by partial or total loss of OTC enzyme activity. It is characterized by elevated plasma ammonia, leading to neurological impairments, coma, and death in the most severe cases. OTCD is managed by combining dietary restr...

Full beskrivning

Sparad:
Bibliografiska uppgifter
I publikationen:Mol Ther Methods Clin Dev
Huvudupphovsmän: De Sabbata, Giulia, Boisgerault, Florence, Guarnaccia, Corrado, Iaconcig, Alessandra, Bortolussi, Giulia, Collaud, Fanny, Ronzitti, Giuseppe, Sola, Marcelo Simon, Vidal, Patrice, Rouillon, Jeremy, Charles, Severine, Nicastro, Emanuele, D’Antiga, Lorenzo, Ilyinskii, Petr, Mingozzi, Federico, Kishimoto, Takashi Kei, Muro, Andrés F.
Materialtyp: Artigo
Språk:Inglês
Publicerad: American Society of Gene & Cell Therapy 2020
Ämnen:
Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC7786024/
https://ncbi.nlm.nih.gov/pubmed/33473356
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.11.005
Taggar: Lägg till en tagg
Inga taggar, Lägg till första taggen!

Liknande verk