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understanding genetic research - Alternating Hemiplegia of
understanding genetic research - Alternating Hemiplegia of

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leber congenital amaurosis - Foundation Fighting Blindness
leber congenital amaurosis - Foundation Fighting Blindness

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EXPERIMENTAL DESIGN is - Universitat de Barcelona

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microarray activity - Blue Valley Schools
microarray activity - Blue Valley Schools

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Bio-Tech - AgriLife Extension County Offices

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Automatically Generating Gene Summaries from Biomedical Literature

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Chapter 19: Human Genetics
Chapter 19: Human Genetics

... Ans: None, all of the children would have one dominant copy and one recessive copy. 20. If one parent has only dominant genes and the other has only recessive genes for a particular dominant genetic disorder, how many of their children should be affected by the disorder? Ans: All of the children, as ...
Transposon insertion frequency distinguishes essential from non
Transposon insertion frequency distinguishes essential from non

... PCR primer contains adapter arm and ...
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Gene therapy



Gene therapy is the therapeutic delivery of nucleic acid polymers into a patient's cells as a drug to treat disease. Gene therapy could be a way to fix a genetic problem at its source. The polymers are either expressed as proteins, interfere with protein expression, or possibly correct genetic mutations.The most common form uses DNA that encodes a functional, therapeutic gene to replace a mutated gene. The polymer molecule is packaged within a ""vector"", which carries the molecule inside cells.Gene therapy was conceptualized in 1972, by authors who urged caution before commencing human gene therapy studies. By the late 1980s the technology had already been extensively used on animals, and the first genetic modification of a living human occurred on a trial basis in May 1989 , and the first gene therapy experiment approved by the US Food and Drug Administration (FDA) occurred on September 14, 1990, when Ashanti DeSilva was treated for ADA-SCID. By January 2014, some 2,000 clinical trials had been conducted or approved.Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers' attention, although as of 2014, it was still largely an experimental technique. These include treatment of retinal disease Leber's congenital amaurosis, X-linked SCID, ADA-SCID, adrenoleukodystrophy, chronic lymphocytic leukemia (CLL), acute lymphocytic leukemia (ALL), multiple myeloma, haemophilia and Parkinson's disease. Between 2013 and April 2014, US companies invested over $600 million in the field.The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers. In 2011 Neovasculgen was registered in Russia as the first-in-class gene-therapy drug for treatment of peripheral artery disease, including critical limb ischemia.In 2012 Glybera, a treatment for a rare inherited disorder, became the first treatment to be approved for clinical use in either Europe or the United States after its endorsement by the European Commission.
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