Gene Therapy
Research from Medical Center Provides New Data about Gene Therapy
Published in Gene Therapy Weekly, January 26th, 2012
2012 JAN 26 -- Research findings, "Hepatorenal correction in murine glycogen storage disease type I with a double-stranded adeno-associated virus vector," are discussed in a new report. According to the authors of a study from Durham, North Carolina, "Glycogen storage disease type Ia (GSD-Ia) is caused by the deficiency of glucose-6-phosphatase (G6Pase). Long-term complications of GSD-Ia include life-threatening hypoglycemia and proteinuria progressing to renal failure."
"A double-stranded (ds) adeno-associated virus serotype 2 (AAV2) vector encoding human G6Pase was pseudotyped with four serotypes, AAV2, AAV7, AAV8, and AAV9, and we evaluated efficacy in...
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Source: Gene Therapy Weekly (2012-01-26)
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