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Nanotechnology in Medicine Krešimir Pavelić Division of Molecular
Nanotechnology in Medicine Krešimir Pavelić Division of Molecular

... and in vivo, and their low intracellular penetration have limited their use in therapeutics. Solutions: to increase antisense stability, to improve cell penetration and also to avoid nonspecific aptameric effects (leading to nonspecific binding of antisense oligonucleotides) the use of particulate c ...
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... Genetic Engineering- manipulating genes for practical purposes Examples 1. Medicine Many medicines, such as the ones used to treat burns, are produced by genetic engineering techniques. 2. Vaccines A person vaccinated with a genetically engineered vaccine will make antibodies against the virus. The ...
Chapter 4: Modern Genetics
Chapter 4: Modern Genetics

... experiment was conducted on four year-old Ashanti Desilva who suffered from ADA deficiency. •Dr. W. French Anderson performed the experimental therapy by infusing white blood cells (with the correct genetic composition) into Ashanti to correct her immunodeficiency. •While the therapy did not yield a ...
highly specific nucleases for gene targeting and
highly specific nucleases for gene targeting and

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Gene cloning

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... fluorescence also tells researchers how much mRNA is being generated by each of the genes that is active. ...
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BIOTEK

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S05 Biotechnology Gene Therapy 1

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... Reduction in total FIX Usage-LEXINGTON, Mass. and AMSTERDAM, Netherlands, July 27, 2016 (GLOBE NEWSWIRE) -- uniQure N.V. (Nasdaq:QURE), a leader in human gene therapy, today announced updated clinical data from its ongoing Phase I/II trial of AMT-060, its proprietary, investigational gene therapy, i ...
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word doc - CSUN.edu

...  ½ of children do not survive into their 20’s  Cause: a deletion of 3 bases in the middle sequences of a protein (removes one a.a.)  The cell membrane cannot pass Cl- ions because the protein channel (CFTR) is destroyed. Sickle Cell Disease  Red blood cells are bent and twisted  Get stuck in th ...
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... will ___________________ or separate, due to physical distance. 4. The further apart the 2 genes are on a chromosome the ________________ likely they will crossover or separate, due to _______________ _____________. B. Linkage Maps 1. To make the map show the ____________ of the genes and the ...
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Intro to Genetics Webquest

... What is DNA? 1) Why is DNA important? 2) What does DNA stand for? 3) Why is DNA called a blueprint? 4) The "twisted ladder" shape of the DNA molecule is called a ...
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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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