CRISPR gene editing

A genome-editing approach that uses CRISPR-guided enzymes to target specific DNA sequences for alteration. Its programmable targeting enables researchers to disrupt, replace, or otherwise modify genetic material.

CRISPR-Cas9: A genome-editing system in which a guide RNA directs the Cas9 enzyme to a matching DNA sequence. Its programmable DNA cuts made CRISPR editing widely accessible.
Sickle cell disease: An inherited blood disorder caused by a variant in the HBB gene that produces abnormal hemoglobin. CRISPR-based treatment can alter blood stem cells to reduce disease symptoms.
Zinc finger nuclease: An engineered DNA-cutting enzyme that combines zinc-finger DNA-binding domains with a nuclease. It can target DNA but requires custom protein engineering rather than a guide RNA.
Off-target effects: Unintended changes caused when a molecular tool acts at sites other than its intended target. Imperfect CRISPR targeting can alter DNA at similar but unintended sequences.
CRISPR: Repeated DNA sequences in many bacteria and archaea that form part of an adaptive defense system against viruses. The editing technology was adapted from this microbial immune system.
Guide RNA: An RNA molecule that directs a CRISPR-associated protein to a complementary nucleic-acid sequence. Its sequence determines where many CRISPR editors bind and act.
Casgevy: A CRISPR-Cas9 therapy that edits a patient's blood stem cells to treat sickle cell disease and beta thalassemia. It became the first approved CRISPR-based therapy in several jurisdictions.
TALEN: A programmable nuclease made from transcription activator-like DNA-binding repeats fused to a DNA-cutting domain. Like CRISPR, it targets chosen sequences, but each target requires engineered proteins.
Mosaicism: The presence of genetically distinct cell populations within one organism derived from a single fertilized egg. Editing after cell division can leave an organism with a mixture of edited and unedited cells.
CRISPR-associated protein: A protein that works with CRISPR sequences in microbial defense or related biotechnological applications. These proteins provide the enzymes that recognize or cut targeted genetic material.

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