mRNA and Gene Editing Offer Hope to Children with Rare Diseases and Mark a Potential Turning Point in Medicine
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Dr. Coller discusses how the science to treat rare diseases now exists, but the real barrier is a regulatory and commercial infrastructure never designed for bespoke, single-patient molecular therapies. He offers the view of gene editing as “molecular surgery” rather than a pharmaceutical drug — a view that could enable the scaling of these breakthroughs beyond one-off miracles.
Read Dr. Coller’s Opinion piece in the New York Times.
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