In Vivo Zinc Finger Nuclease-mediated Targeted Integration of a Glucose-6-phosphatase Transgene Promotes Survival in Mice With Glycogen Storage Disease Type IA

Glycogen storage disease type Ia (GSD Ia) is caused by glucose-6-phosphatase (G6Pase) deficiency in association with severe, life-threatening hypoglycemia that necessitates lifelong dietary therapy. Here we show that use of a zinc-finger nuclease (ZFN) targeted to the ROSA26 safe harbor locus and a...

Full beskrivning

Sparad:
Bibliografiska uppgifter
I publikationen:Mol Ther
Huvudupphovsmän: Landau, Dustin J, Brooks, Elizabeth Drake, Perez-Pinera, Pablo, Amarasekara, Hiruni, Mefferd, Adam, Li, Songtao, Bird, Andrew, Gersbach, Charles A, Koeberl, Dwight D
Materialtyp: Artigo
Språk:Inglês
Publicerad: Nature Publishing Group 2016
Ämnen:
Original Article
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4886939/
https://ncbi.nlm.nih.gov/pubmed/26865405
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.35
Taggar: Lägg till en tagg
Inga taggar, Lägg till första taggen!

Liknande verk