Gene therapy for SCAD

Posted by & filed under Part 12: LIPIDS.

Hum Gene Ther. 2006 Jan;17(1):71-80.

Systemic correction of a fatty acid oxidation defect by intramuscular injection
of a recombinant adeno-associated virus vector.

Conlon TJ, Walter G, Owen R, Cossette T, Erger K, Gutierrez G, Goetzman E,
Matern D, Vockley J, Flotte TR.

In this article by a group from Pittsburg, proof of principle for gene therapy of a fatty acid oxidation defect (SCAD deficiency) was provided using rAAV in a murine model.
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Philippe Campeau, MD
Resident in Medical Genetics at McGill University
OMMBID Blog Administrator

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