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Bonnie Steinbock University at Albany (emerita)
Bonnie Steinbock University at Albany (emerita)

... Editas hopes to use CRISPR in a clinical trial by 2017 to treat a rare form of blindness, Leber congenital amaurosis ...
DNA TECHNOLOGY - Mount Mansfield Union High School
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... continued Cloning, continued • The first clone made from an adult mammal was made using a process called somatic-cell nuclear transfer (SCNT). • Although scientists have successfully cloned many kinds of animals, only a few of the cloned offspring have survived for long. • Some problems with cloning ...
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Assessment Questions - Teach Genetics (Utah)

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Mendelian Genetics
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... A human baby boy inherits a recessive allele from his mother. In which circumstance would he most likely show the trait coded for by the recessive allele? A. The baby inherits the dominant allele from his father. B. The allele is on an autosomal chromosome and the baby is a twin. C. The allele is on ...
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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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