Articles in topic: Gene therapy
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CRISPR gene editing: treatment of hypertriglyceridemia CTX310
AAV gene therapy for HoFH: cholesterol reduction by 97.5% in clinical trial
CRISPR vision restoration: FDA approval for testing
CRISPR-Cas3 treatment of Duchenne muscular dystrophy: deletion of exons 45-55
Intellia CRISPR therapy: phase 3 data on edema — breakthrough
Intellia CRISPR therapy: FDA filing for lonvo-z for HAE
AAV9 gene therapy for SMA: long-term efficacy study
ATF2 gene: key CRISPR target for neuron rescue
AI-designed viral vectors: 50-fold increase in gene delivery to the brain
Gene therapy OPGx-LCA5 accepted into FDA RDEP program
Casgevy: CRISPR therapy approved by FDA | Breakthrough in treatment
