Canine blood disorder treated with gene therapy
Factor VII deficiency is a rare inherited bleeding disorder that causes mild to moderate blood clotting, and impacts beagles, Airedales, Alaskan Klee kais, giant schnauzers, and Scottish deerhound breeds, according to the University of California, Davis, Veterinary Genetics Lab. A new study suggests a remedy.
Researchers from the University of North Carolina (UNC) and the Raymond G. Perelman Center for Cellular and Molecular Therapeutics (CCMT) at The Children’s Hospital of Philadelphia (CHOP) have used a single injection of gene therapy to correct the factor VII deficiency in dogs.
The study was published on Dec. 23, 2015, in Blood.
“We developed a unique animal model of this disease after identifying dogs with naturally occurring factor VII deficiency,” said Paris Margaritis, DPhil, and the study’s lead author. “Our investigations enabled us to design the corrective gene to insert into our virus vector in the current study.”
The researchers characterized factor VII in four individual dogs. Using the AAV vectors supplied by Margaritis, researchers injected the dogs with varying dosages and monitored their health outcomes and biological markers over several years.
Based on kidney function, liver function, and blood measurements in the dogs, the treatment was safe, and did not elicit unwanted immune responses.
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