Antibody Gene Therapy Shows Promise in Mouse Models of Huntington’s Disease

Researchers at the California Institute of Technology have shown that a highly specific intrabody (an antibody fragment that works against a target inside a cell) is capable of stalling the development of Huntington's disease in a variety of mouse models. "Gene therapy in these models successfully attenuated the symptoms of Huntington's disease and increased life span," noted Dr. Paul Patterson, the senior author of the study. The intrabody, called Happ1, targets an amino acid sequence unique to the huntingtin protein that is rich in the amino acid proline. Because of this, the action of Happ1 is expected to be extremely specific. "Our studies show that the use of intrabodies can block the parts of mutant huntingtin that cause its toxicity without affecting the wild type, or normal, huntingtin—or any other proteins," said Dr. Patterson. In other words, he said, this approach has the potential to be the kind of "silver-bullet therapy" that many medical researchers look for. With regard to future work, Dr. Patterson said, “we need to improve the efficacy of the intrabody, and we need to build a viral vector that can be controlled—induced and turned off—in case of unexpected side effects. This is a general goal shared by all types of experimental gene therapies." This work was published in the October 28 issue of the Journal of Neuroscience. [Press release] [Journal of Neuroscience abstract]
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