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 genetic cause, making it especially relevant to hereditary disorders arising from defined mutation…
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 genetic cause, making it especially relevant to hereditary disorders arising from defined mutations in single genes or coagulation and metabolic pathways. Approaches include adding a functional copy of a defective gene, using gene-editing tools such as CRISPR to repair or disrupt specific sequences, and employing RNA-based methods like small interfering RNA to reduce expression of harmful gene products. Delivery is a central challenge: corrective sequences must reach the appropriate tissue and cells, often via viral or nanoparticle carriers, and achieve durable, regulated expression without unwanted effects. Research spans inherited bleeding disorders, lysosomal storage and metabolic conditions, neurodegenerative diseases, and applications extending to cardiovascular failure and metabolic disease such as type 2 diabetes. Work in this area also connects to the molecular characterization of disease-causing mutations and to ethical questions raised by genetic medicine, particularly when interventions are heritable or experimental. Effective gene therapy depends on precise mutation identification, safe and targeted delivery, controlled expression, and rigorous evaluation of efficacy and long-term safety.
11 peer-reviewed articles, ranked by relevance. Each links to its DOI.
The 11 articles above have been cited 15 times in the scholarly literature. Citation data via OpenAlex and Crossref, updated Jun 2026.
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