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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 is the genetic engineering of humans, generally by replacing defective genes with effective ones. Clinical research using somatic gene therapy has been conducted with several diseases, including X-linked SCID, [93] chronic lymphocytic leukemia (CLL), [94] [95] and Parkinson's disease. [96]
The treatment of genetic disorders is an ongoing battle, with over 1,800 gene therapy clinical trials having been completed, are ongoing, or have been approved worldwide. [38] Despite this, most treatment options revolve around treating the symptoms of the disorders in an attempt to improve patient quality of life .
A number of recent clinical trials have suggested that gene therapy could help treat diseases of the eye that cause vision loss. This Special Feature explores the current evidence and outstanding ...
The Food and Drug Administration on Friday approved Pfizer’s treatment for a rare genetic bleeding disorder, making it the company’s first-ever gene therapy to win clearance in the U.S.. The ...
Student Zoe Davis, 20, was just weeks into her junior year when she landed back in the hospital with severe sickle cell pain earlier this month. She knows new gene therapies may provide long-term ...
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