Boston Children’s and Broad Institute launch rare disease center

A collaboration among the Broad Institute, Boston Children’s Hospital, and Maine’s Jackson Laboratory has been announced to develop gene therapies for rare diseases and make them more accessible. The initiative, through the non-profit Center for Therapeutic Genetics, aims to create shared treatment platforms, including gene-editing methods, to streamline therapy development and delivery. The focus is on treating children with rare genetic epilepsies, supported by federal funding, with the goal of standardizing gene therapies to resemble routine clinical procedures and improve patient access.

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