Nature

Rescue administration of a helper-dependent adenovirus vector with long-term efficacy in dogs with glycogen storage disease type Ia

Glycogen storage disease type Ia (GSD-Ia) stems from glucose-6-phosphatase (G6Pase) deficiency and causes hypoglycemia, hepatomegaly, hypercholesterolemia and lactic acidemia. Three dogs with GSD-Ia ...
Nature

Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B

Hemophilia B, the deficiency in clotting factor IX (FIX), is an X-linked recessive disorder that occurs in about one in 25 000 males in the US. Of these patients, approximately 40% are characterized ...
Business Insider

Co-administration of AAV Vectors with SVP-Rapamycin Enables Vector Re-administration in Pre-clinical Gene Therapy Study Published in Nature Communications by Généthon and ...

Represents a potentially powerful strategy to modulate vector immunogenicity and may open new therapeutic avenues for AAV vector-mediated gene transfer EVRY, France and WATERTOWN, Mass., Oct. 05, 2018 ...