CRISPR-Cas9 Gene Editing

Category: technique

A revolutionary gene editing technology that uses guide RNA to direct the Cas9 nuclease to specific genomic locations for precise DNA modification.

Mechanism Detail

CRISPR-Cas9 creates double-strand breaks at targeted genomic loci. Cells repair these breaks via non-homologous end joining (NHEJ) for gene disruption, or homology-directed repair (HDR) for precise edits. Base editors and prime editors offer single-nucleotide precision without double-strand breaks.

Clinical Status

Casgevy (exagamglogene autotemcel) FDA-approved for sickle cell disease (2023). Multiple cancer and neurological trials underway.

Relevant Diseases

• ALS
• Stage IV Cancer
• Parkinson's Disease

Relevant Therapies

• Gene Therapy

Related Terms

• Base editing
• Prime editing
• Guide RNA
• AAV vectors