Tag: CRISPR

Medical illustration showing CRISPR gene editing process for hereditary angioedema treatment
New StudiesResearch Digest

First In Vivo CRISPR Gene Editing Treatment Shows Promise for Rare Blood Disorder

First clinical trial of in vivo CRISPR gene editing for hereditary angioedema shows promising results. The investigational therapy targets genetic…

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GMJ Research Desk
Medical illustration showing CRISPR gene editing process for blood disorders treatment
Clinical UpdatesNew StudiesPracticeResearch Digest

CRISPR Gene Therapy Shows Promise for Children with Sickle Cell Disease and Beta-Thalassemia

CRISPR gene therapy exa-cel achieved 95% efficacy in eliminating blood transfusions for children with beta-thalassemia and sickle cell disease. New…

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GMJ Practice Desk
Illustration of small-molecule drugs controlling genome editing tools for safer gene therapy
New StudiesResearch Digest

Small-Molecule Drugs Enable Safer, More Precise Therapeutic Genome Editing

New research shows small-molecule drugs can precisely control therapeutic genome editing, achieving 85% improved accuracy while reducing off-target effects by…

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GMJ Research Desk

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