Gene Therapy Method to Target Kidney Cells Developed

First examination of adeno-associated virus (AAV) subtypes for delivering and transferring (or "transducing") genetic material to the kidney has been examined by a team led by Benjamin D. Humphreys MD, PhD (Washington University in St. Louis). The findings, which appear in an upcoming issue of the Journal of the American Society of Nephrology (JASN), could have important implications for the treatment of chronic kidney disease (CKD).

Almost 50 years after it was first proposed, gene therapy is now entering into regular clinical use. The most common method uses adeno-associated virus (AAV) to deliver genetic material to target cells, but no AAV has been capable of doing so in the kidney.

Of 6 AAVs tested, a synthetic AAV called Anc80 showed specific and high efficiency for transducing different kidney cell types in both mice and humans. Anc880 was also successfully used in gene therapy strategies to treat mice with kidney scarring.

The findings may be useful in therapeutic approaches to treat CKD, a condition that affects10% of the population worldwide. "CKD is an enormous problem and there has been little innovation and no significant therapies in decades. Our discovery shows that genetic material can be delivered to kidney cells that play a key role in CKD," said Dr. Humphreys. "Future gene therapy approaches could deliver genes that slow or reverse the growth of these cells in CKD."

Source-Eurekalert