In a highly anticipated article, FDA officials Vinay Prasad, M.D., and Martin Makary, M.D., outlined a novel regulatory pathway that could trigger a seismic shift in how bespoke gene editing therapies ...
Many neurological diseases are caused by pathogenic unstable intragenic trinucleotide repeat (TNR) expansions, such as the GAA trinucleotide repeat within the FXN gene in Friedreich ataxia (FRDA) and ...
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