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0 -0.000459347726228664 0.00413412953605879 0.00459347726228762 0.00505282498851644 0.00137804317868632 -0.00528249885163061 -0.0105649977032612
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Gene Editing Pioneer David Liu: Fixing Baby's Rare Disease Heralds New Medical Era [Forbes]

Beam Therapeutics Inc. (BEAM) 
Company Research Source: Forbes
created a customized gene editing therapy to correct KJ's rare genetic mutation, which made his body unable to properly process protein. Without this personalized treatment, his future looked bleak: the possibility of progressive brain damage due to high ammonia levels his body could not remove. But after three infusions containing billions of gene editors delivered to his liver to correct the mutation, today baby KJ appears to be doing well and developing normally. At a conference last month hosted by STAT News, the adorably plump boy squirmed in a tiny tuxedo. I recently had the privilege of speaking to one of key scientists behind this breakthrough: Dr. David Liu of the Broad Institute, whose lab is credited with developing both prime editing and base editing, which is the technology used to treat baby KJ. In an hour-long interview, Liu offered insights on this hopeful time in medical history, the scientific frontiers he and his colleagues continue to push, and the bottlenecks Show less Read more
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