【Animal Modeling】-Biological distribution of AAV gene transfer vector in non-human primates

  New Rochelle, New York, January 15, 2021-A new method can be used to measure the biodistribution of adeno-associated virus gene transfer vectors in non-human primates. This method quantitatively detects systemic and organ-specific AAV capsids within 1 to 72 hours after administration. "The AAV capsid is labeled with I-124 and is delivered by two routes of administration: intravenously and directly into the cerebrospinal fluid (CSF). Biodistribution was measured by quantitative positron emission tomography (PET) at 1, 24, 48, and 72 hours after the administration of AAV. Two AAV vectors-AAVrsh.10 and AAV9 were compared. Ronald Crystal said: "After intravenous administration, both carriers behave similarly, mainly in the liver and to a lesser extent the heart. Neither of them was detected in the brain. Veins. Both carriers for internal administration are also distributed in the vertebrae." Weill Cornell School of Medicine, and co-author. Approximately 50% is dispersed throughout the body, and part is dispersed in skeletal muscle.

   After injection into the cerebrospinal fluid, the half-life of the labeled capsid is about 10 hours, which indicates that it may slowly spread to the brain.

   Compared with non-immunized animals, the biodistribution of the spleen in animals with existing immunity increased 10 times. Human Gene Therapy Editor-in-Chief Terence R. Flotte (Terence R. Flotte), MD, Celia and Professor Isaac Haidak said: "PET imaging is tracking biological A powerful tool for distribution, which is a key feature that affects the safety and effectiveness of gene therapy." University of Massachusetts Medical Education and Provost, Provost and Executive Vice President.