Gene therapy is a therapeutic strategy that treats disease by introducing, correcting, silencing, or replacing genetic material within a patient's cells. Rather than addressing symptoms alone, it targets the underlying molecular defect, whether by supplying a functional copy of a gene, editing a pathogenic sequence,…
Gene therapy is a therapeutic strategy that treats disease by introducing, correcting, silencing, or replacing genetic material within a patient's cells. Rather than addressing symptoms alone, it targets the underlying molecular defect, whether by supplying a functional copy of a gene, editing a pathogenic sequence, or suppressing the activity of a harmful transcript. Approaches are commonly grouped by mechanism and delivery: gene addition, gene editing using tools such as CRISPR, and RNA-based silencing through small interfering RNA, alongside the viral and non-viral vectors, including nanoparticle carriers, used to reach target tissues. The field spans a broad range of conditions. Investigators explore correction of inherited metabolic and lysosomal storage disorders, modulation of coagulation factor deficiencies, and intervention in cardiovascular and metabolic disease, while in oncology efforts focus on the somatic alterations and signaling pathways that drive tumor growth. Effective gene therapy depends on precise targeting, durable and regulated expression, and acceptable safety, and its development draws on genomic and proteomic characterization of disease mechanisms and candidate genes. The discipline also raises distinctive ethical considerations regarding germline modification, access, and the boundaries of genetic medicine. As delivery and editing technologies mature, gene therapy continues to move from proof of concept toward durable, mechanism-based treatment of previously intractable disorders.
12 peer-reviewed articles, ranked by relevance. Each links to its DOI.
The 12 articles above have been cited 19 times in the scholarly literature. Citation data via OpenAlex and Crossref, updated Jun 2026.
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