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Making the Grade: Testing for Human Genetic Disorders
Making the Grade: Testing for Human Genetic Disorders

... possibility of a mistaken diagnosis raises questions regarding the distribution of the risks and benefits of an imperfect testing procedure. 9 Nevertheless, this Article assumes, for the purposes of this discussion, that the tests are flawless, since it is important to look at the ethical issues ari ...
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MBG 304 Molecular Genetics of Eukaryotes (3+0)3

... evolutionary genetics, and the genetics of common diseases. Survey of human genetic conditions with an emphasis on the underlying molecular biology. MBG 324 Plant Molecular Biology and Genetics (3+0)3 This course emphasizes genetic transformation methodology, gene expression systems and strategies f ...
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Draft breeding policy - Balinese Breed Advisory Committee

... helpful to know about the ancestors of the cats when trying to predict the result of a mating. For example a black cat with a blue mother will carry dilute and so can produce blue offspring if mated to a blue, or to another carrier. But, though from the ancestry one can determine when a recessive al ...
Posttest Questions, Answers, and Rationale 1. All of the following
Posttest Questions, Answers, and Rationale 1. All of the following

... Rationale: The goal of vasoconstrictor therapy is to improve urine output and decrease SCr. The initial goal for titration is to increase the mean arterial pressure by > 10 mm Hg. An unintentional consequence of treating HRS is that improving renal function decreases a patient’s MELD score, which lo ...
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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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