Foundation, medical schools partner on gene therapy for H-ABC

By The Science Advisory Board staff writers

October 13, 2020 -- The nonprofit organization Foundation to Fight H-ABC has signed an agreement with the University of Massachusetts Medical School and Yale University to sponsor research aimed at using gene therapy to treat hypomyelination with atrophy of the basal ganglia and cerebellum (H-ABC), a rare degenerative children's disease.

There is currently no cure for H-ABC, a disabling and life-threatening genetic disorder caused by a mutation in the TUBB4A gene that affects certain parts of the brain that control movement. In the new collaborative research effort, Dr. Guangping Gao of the University of Massachusetts and Dr. Karel Liem of Yale will team up to develop adeno-associated virus (AAV) vectors to silence or outcompete the mutated TUBB4A gene, according to the foundation.

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