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Gene Therapy

Definition
AI-generated

Gene therapy delivers genetic material (or edits the genome) to treat or prevent disease—using viral or non-viral vectors, ex vivo cell engineering, or targeted nucleases—with the goal of durable correction or modulation of gene function.

Topics
Precision medicine
Related
Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Gene Genome-Wide Association Study (GWAS)

Why it matters in GWAS

Human genetic associations nominate causal genes and cell types for intervention; Mendelian disease genes are classic gene-therapy targets. Germline editing raises distinct ethics and safety issues compared with somatic therapy, and long-read or WGS characterization of individuals can inform vector design and immune responses.

Example usage

"The program tracked long-term outcomes after ex vivo gene therapy for the monogenic disorder."

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) Gene Genome-Wide Association Study (GWAS) Precision Medicine
Related-term network

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Node order. The current term is first. Next come every page linked from Related terms, in the same top-to-bottom order as that list. Then new terms appear in two-hop layers: for each of those direct neighbors (in that same order), their related terms are appended if not already listed. A third pass does the same for two-hop nodes. The walk stops when a fixed maximum number of nodes is reached.

References

  • Dunbar CE, et al. (2018). Gene therapy comes of age. Science.
← Gene Regulatory Network Inference Genealogy →

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