What is Gene Therapy? | FDA - U.S. Food and Drug Administration
Human gene therapy is the administration of genetic material to modify or manipulate the expression of a gene product or to alter the biological properties of living cells for therapeutic use.
Gene therapy - Mayo Clinic
2024年4月23日 · Gene therapy aims to fix a faulty gene or replace it with a healthy gene to try to cure disease or make the body better able to fight disease. It holds promise as a treatment for a wide range of diseases, such as cancer, cystic fibrosis, …
Gene therapy - Wikipedia
Gene therapy approaches to replace a faulty gene with a healthy gene have been proposed and are being studied for treating some genetic diseases. As of 2017, 11.1% of gene therapy clinical trials targeted monogenic diseases.
Gene Therapy Definition, Pros & Cons - Cleveland Clinic
Gene therapy is a medical approach that uses genetic material to prevent and treat disease. The technique allows healthcare providers to treat certain conditions by changing your genetic makeup instead of using traditional treatment methods like medication and surgery.
Gene therapy | Description, Uses, Examples, & Safety Issues
1 天前 · Gene therapy, introduction of a normal gene into an individual’s genome in order to repair a mutation that causes a genetic disease. Human gene therapy has been attempted on somatic (body) cells for diseases such as cystic fibrosis and cancer.
Gene Therapy - National Human Genome Research Institute
2024年11月21日 · Gene therapy is a technique that uses a gene(s) to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, or by replacing a defective or missing gene in …
What is gene therapy? - MedlinePlus
2022年2月28日 · Gene therapy is a medical approach that treats or prevents disease by correcting the underlying genetic problem instead of using drugs or surgery.
How Does Gene Therapy Work? Types, Uses, Safety - Healthline
2023年1月21日 · Gene therapy can treat certain genetic conditions by editing diseased cells. Learn about its uses, safety, and potential downsides here.
How Gene Therapy Can Cure or Treat Diseases | FDA
2022年7月28日 · The FDA has approved gene therapies for cancer, rare diseases. Scientists have worked for decades on ways to modify or replace genes to treat, cure, or prevent illness.
A primer to gene therapy: Progress, prospects, and problems
In this review, we summarize the features of state‐of‐the‐art viral vector systems and the corresponding gene‐centered therapies they seek to deliver. Moreover, a brief summary is also given on emerging gene editing approaches built on CRISPR‐Cas9 nucleases and, more recently, nickases, including base editors and prime editors.