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Phase 3 CRISPR trial shows durable benefit for hereditary angioedema
One post describes CRISPR-mediated in vivo gene editing that inactivated the prekallikrein gene in liver cells.
TLDR
A post describes a phase 3 trial of in vivo CRISPR editing that inactivated the prekallikrein gene in liver cells, with what it calls impressive and durable benefit for hereditary angioedema. It also points to a separate NEJM report of a paraspinal tumor after AAV gene therapy, which the post says was caused by integrated AAV sequences driving GLI1 expression. The author calls for careful design, supervision and monitoring of molecular-medicine treatments.
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