Zobrazeno 1 - 2
of 2
pro vyhledávání: '"Kamal K E, Gadalla"'
Publikováno v:
PLoS ONE, Vol 9, Iss 11, p e112889 (2014)
Rett syndrome (RTT) is a genetic disorder characterized by a range of features including cognitive impairment, gait abnormalities and a reduction in purposeful hand skills. Mice harbouring knockout mutations in the Mecp2 gene display many RTT-like ch
Externí odkaz:
https://doaj.org/article/fbd81503313d477bb13f07d3700029f2
Autor:
Sarah E, Sinnett, Ralph D, Hector, Kamal K E, Gadalla, Clifford, Heindel, Daphne, Chen, Violeta, Zaric, Mark E S, Bailey, Stuart R, Cobb, Steven J, Gray
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Intravenous administration of adeno-associated virus serotype 9 (AAV9)/hMECP2 has been shown to extend the lifespan of Mecp2−/y mice, but this delivery route induces liver toxicity in wild-type (WT) mice. To reduce peripheral transgene expression,