Gene therapy
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 Gene therapy using an Adenovirus vector. A new gene is inserted into an adenovirus. If the treatment is successful, the new gene will make functional protein to treat a disease. |
Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene in order to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease.
- Related: Antisense therapy, DNA, Full Genome Sequencing, Genetic engineering, Life extension, Pharmacological gene therapy, Predictive Medicine, Technology assessment, Therapeutic gene modulation
| Gene Therapy: Molecular Bandage? Gene Therapy: Molecular Bandage? learn.genetics.utah.edu/content/tech/genetherapy/index.html - Web |
| The American Society of Gene & Cell Therapy The American Society of Gene & Cell Therapy www.asgt.org - Web |
| The European Society of Gene & Cell Therapy The European Society of Gene & Cell Therapy www.esgct.eu - Web |
Average relevance
| Research Group at Cambridge, UK working on overcom... Research Group at Cambridge, UK working on overcoming current hurdles to successful gene therapy www.cheng.cam.ac.uk/research/groups/biosci/index.html - Web |
| Council for Responsible Genetics Council for Responsible Genetics www.gene-watch.org - Web |
| Molecular Medicine and Gene Therapy at Lund Univer... Molecular Medicine and Gene Therapy at Lund University www.med.lu.se/labmedlund/molekylaer_medicin_och_genterapi - Web |
| Gene Therapy Frees β-Thalassemia Patient From Tran... Gene Therapy Frees β-Thalassemia Patient From Transfusions www.medscape.com/viewarticle/728656 - Web |
| Clinical Trial at Sloan Kettering Clinical Trial at Sloan Kettering clinicaltrials.gov/.../NCT01639690?term=lentiviral+thalassemia&rank=1 - Web |
| Stem Cell Therapy Trial Offers Hope Stem Cell Therapy Trial Offers Hope www.mskcc.org/.../launch-stem-cell-therapy-trial-offers-hope-patients-inherited-blood-disorder - Web |
