Crispr
3 articles
Replacement, Editing, and the Two Routes Into the Eye: How Gene Therapy Is Being Built for Inherited Retinal Diseases
Gene therapy for inherited retinal diseases employs two main strategies: gene replacement or gene editing, delivered via subretinal or intravitreal injection. While Luxturna offers a successful model for LCA, ongoing research for conditions like achromatopsia highlights the complexities and challenges in developing effective treatments.
CRISPR's Expanding Role: Beyond Gene Editing in Diagnostics and Epigenetic Control
CRISPR technology has evolved significantly beyond gene editing, now offering powerful applications in rapid disease diagnostics and precise epigenetic control of gene expression without altering DNA sequences. Its versatility continues to expand into areas like RNA editing and live-cell imaging, promising broad biotechnological advancements.
What Are CRISPR Gene Editing Mechanisms and Medical Applications?
An 18-year-old with a rare immune disorder recently received the first-ever prime editing treatment.