Daily Archives: July 28, 2015

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. The first gene therapy experiment approved by the US Food and Drug Administration (FDA) occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID.[1] By January 2014, some 2,000 clinical trials had been conducted or approved.[2]

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.[3] These include treatment of retinal disease Leber's congenital amaurosis,[4][5][6][7]X-linked SCID,[8] ADA-SCID,[9][10]adrenoleukodystrophy,[11]chronic lymphocytic leukemia (CLL),[12]acute lymphocytic leukemia (ALL),[13]multiple myeloma,[14]haemophilia[10] and Parkinson's disease.[15] Between 2013 and April 2014, US companies invested over $600 million in the field.[16]

The first commercial gene therapy, Gendicine, was approved in China in 2003 for the treatment of certain cancers.[17] 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.[3][18]

Following early advances in genetic engineering of bacteria, cells and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered replacing or disrupting defective genes.[19] Scientists focused on diseases caused by single-gene defects, such as cystic fibrosis, haemophilia, muscular dystrophy, thalassemia and sickle cell anemia. Glybera treats one such disease, caused by a defect in lipoprotein lipase.[18]

DNA must be administered, reach the damaged cells, enter the cell and express/disrupt a protein.[20] Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered virus to deliver the DNA into a chromosome.[21][22]Naked DNA approaches have also been explored, especially in the context of vaccine development.[23]

Generally, efforts focused on administering a gene that causes a needed protein to be expressed. More recently, increased understanding of nuclease function has led to more direct DNA editing, using techniques such as zinc finger nucleases and CRISPR. The vector incorporates genes into chromosomes. The expressed nucleases then "edit" the chromosome. As of 2014 these approaches involve removing cells from patients, editing a chromosome and returning the transformed cells to patients.[24]

Other technologies employ antisense, small interfering RNA and other DNA. To the extent that these technologies do not alter DNA, but instead directly interact with molecules such as RNA, they are not considered "gene therapy" per se.[citation needed]

Gene therapy may be classified into two types:

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Gene therapy - Wikipedia, the free encyclopedia

Human Genetic Engineering - A Hot Issue! Human genetic engineering is a hot topic in the legislative and executive branches of the U.S. government. Time will tell how committed the United States will be regarding the absolute ban on human cloning.

Human Genetic Engineering - Position of the U.S. Government Human genetic engineering has made its way to Capitol Hill. On July 31, 2001, the House of Representatives passed a bill which would ban human cloning, not only for reproduction, but for medical research purposes as well. The Human Cloning Prohibition Act of 2001, sponsored by Rep. Weldon (R-fL) and co-sponsored by over 100 Representatives, passed by a bipartisan vote of 265-to-162. The Act makes it unlawful to: "1) perform or attempt to perform human cloning, 2) participate in an attempt to perform cloning, or 3) ship or receive the product of human cloning for any purpose." The Act also imposes penalties of up to 10 years imprisonment and no less than $1,000,000 for breaking the law. The same bill, sponsored by Sen. Brownback (R-kS), is currently being debated in the Senate.

The White House also opposes "any and all attempts to clone a human being; [they] oppose the use of human somatic cell nuclear transfer cloning techniques either to assist human reproduction or to develop cell or tissue-based therapies."

Human Genetic Engineering - The Problems There are many arguments against human genetic engineering, including the established safety issues, the loss of identity and individuality, and human diversity. With therapeutic cloning, not only do the above issues apply, but you add all the moral and religious issues related to the willful killing of human embryos. Maybe the greatest concern of all is that man would become simply another man-made thing. As with any other man-made thing, the designer "stands above [its design], not as an equal but as a superior, transcending it by his will and creative prowess." The cloned child will be dehumanized. (See, Leon Kass, Preventing a Brave New World: Why we should ban human cloning now, New Republic Online, May 21, 2001.)

Human Genetic Engineering - A Final Thought Human genetic engineering leads to man usurping God as the almighty creator and designer of life. No longer will a child be considered a blessing from God, but rather, a product manufactured by a scientist. Man will be a created being of man. However, man was always intended to be a created being of God, in His absolute love, wisdom and glory.

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Human Genetic Engineering - Popular Issues

David King

The main debate around human genetics currently centres on the ethics of genetic testing, and possibilities for genetic discrimination and selective eugenics. But while ethicists and the media constantly re-hash these issues, a small group of scientists and publicists are working towards an even more frightening prospect: the intentional genetic engineering of human beings. Just as Ian Wilmut presented us with the first clone of an adult mammal, Dolly, as a fait accompli, so these scientists aim to set in place the tools of a new techno-eugenics, before the public has ever had a chance to decide whether this is the direction we want to go in. The publicists, meanwhile are trying to convince us that these developments are inevitable. The Campaign Against Human Genetic Engineering, has been set up in response to this threat.

Currently, genetic engineering is only applied to non-reproductive cells (this is known as 'gene therapy') in order to treat diseases in a single patient, rather than in all their descendants. Gene therapy is still very unsuccessful, and we are often told that the prospect of reproductive genetic engineering is remote. In fact, the basic technologies for human genetic engineering (HGE) have been available for some time and at present are being refined and improved in a number of ways. We should not make the same mistake that was made with cloning, and assume that the issue is one for the far future.

In the first instance, the likely justifications of HGE will be medical. One major step towards reproductive genetic engineering is the proposal by US gene therapy pioneer, French Anderson, to begin doing gene therapy on foetuses, to treat certain genetic diseases. Although not directly targeted at reproductive cells, Anderson's proposed technique poses a relatively high risk that genes will be 'inadvertently' altered in the reproductive cells of the foetus, as well as in the blood cells which he wants to fix. Thus, if he is allowed to go ahead, the descendants of the foetus will be genetically engineered in every cell of their body. Another scientist, James Grifo of New York University is transferring cell nuclei from the eggs of older to younger women, using similar techniques to those used in cloning. He aims to overcome certain fertility problems, but the result would be babies with three genetic parents, arguably a form of HGE. In addition to the two normal parents, these babies will have mitochondria (gene-containing subcellular bodies which control energy production in cells) from the younger woman.

Anderson is a declared advocate of HGE for medical purposes, and was a speaker at a symposium last year at UCLA, at which advocates of HGE set out their stall. At the symposium, which was attended by nearly 1,000 people, James Watson, of DNA discovery fame, advocated the use of HGE not merely for medical purposes, but for 'enhancement': 'And the other thing, because no one really has the guts to say it, I mean, if we could make better human beings by knowing how to add genes, why shouldn't we do it?'

In his recent book, Re-Making Eden (1998), Princeton biologist, Lee Silver celebrates the coming future of human 'enhancement', in which the health, appearance, personality, cognitive ability, sensory capacity, and life-span of our children all become artifacts of genetic engineering, literally selected from a catalog. Silver acknowledges that the costs of these technologies will limit their full use to only a small 'elite', so that over time society will segregate into the "GenRich" and the "Naturals":

"The GenRich - who account for 10 percent of the American population - all carry synthetic genes... that were created in the laboratory ...All aspects of the economy, the media, the entertainment industry, and the knowledge industry are controlled by members of the GenRich class...Naturals work as low-paid service providers or as labourers, and their children go to public schools... If the accumulation of genetic knowledge and advances in genetic enhancement technology continue ... the GenRich class and the Natural class will become...entirely separate species with no ability to cross-breed, and with as much romantic interest in each other as a current human would have for a chimpanzee."

Silver, another speaker at the UCLA symposium, believes that these trends should not and cannot be stopped, because to do so would infringe on liberty.

Most scientists say that what is preventing them from embarking on HGE is the risk that the process will itself generate new mutations, which will be passed on to future generations. Official scientific and ethical bodies tend to rely on this as the basis for forbidding attempts at HGE, rather than any principled opposition to the idea.

In my view, we should not allow ourselves to be lulled into a false sense of security by this argument. Experience with genetically engineered crops, for example, shows that we are unlikely ever to arrive at a situation when we can be sure that the risks are zero. Instead, when scientists are ready to proceed, we will be told that the risks are 'acceptable', compared to the benefits. Meanwhile, there will be people telling us loudly that since they are taking the risks with their children, we have no right to interfere.

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Human Genetics Alert - The Threat of Human Genetic Engineering