Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia.
Koeberl DD, Sun BD, Damodaran TV, Brown T, Millington DS, Benjamin DK, Bird A,
Schneider A, Hillman S, Jackson M, Beaty RM, Chen YT.
Gene Ther. 2006 May 4
This group describes the IV administration of a pseudotyped AAV8 vector for the correction of glycogen storage disease 1a in a mouse model. Partial biochemical correction was sustained up to 7 months after the administration.
For more information on GSD-1a, please see chapter 71.
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Philippe Campeau, MD
Resident in Medical Genetics at McGill University
OMMBID Blog Administrator