Daily Archives: March 11, 2014

Genetic engineering got wrong
Frankenstein version.

By: jacmalayter

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Genetic engineering got wrong - Video

PUBLIC RELEASE DATE:

11-Mar-2014

Contact: Jennifer Quigley jquigley@liebertpub.com 914-740-2100 x2149 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, March 11, 2014Several young children suffering from a severe degenerative genetic disease received injections of therapeutic genes packaged within a noninfectious viral delivery vector. Safety, tolerability, and efficacy results from this early stage clinical trial are reported in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website.

Marc Tardieu, Universit Paris-Sud and INSERM, and a team of international researchers administered the adeno-associated viral (AAV) vector carrying a normal copy of the N-sulfoglycosamine sulfohydrolase (SGSH) gene into the brains of four children affected by mucopolysaccharidosis type IIIA (MPSIIIA), an inherited lysosomal storage disease in which the SGSH gene is defective. The AAV vector also delivered a sulfatase-modifying factor (SUMF1), needed to activate the SGSH protein.

In addition to measures of toxicity, adverse events, and tolerability, the researchers evaluated the children for brain shrinkage (a characteristic of MPSIIIA) and for changes in behavior, attention, sleep, and cognitive benefit. They describe their findings in the article "Intracerebral administration of AAV rh.10 carrying human SGSH and SUMF1 cDNAs in children with MPSIIIA disease: results of a phase I/II trial."

"This is an important new approach for treating CNS manifestations of lysosomal storage diseases that could be applied across a wide array of disorders," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly and focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

Originally posted here:
Gene therapy for lysosomal storage disease shown to be safe and well tolerated

March 12, 2014, 12:13 a.m.

A new technique looks set to soon allow consenting parents to have a child who will not suffer from a serious disability.

Parents in the UK look set to become the first in the world to use a radical IVF technique that some critics have condemned as eugenic engineering. If approved by parliament, so called "three person IVF" could be available on the National Health Service as early as next year.

Supporters hail the technique as a cure for the debilitating and incurable diseases caused by defective genetic material in a part of the mother's egg cell called the mitochondria. It involves implanting the nucleus of a woman's egg into another woman's egg cell which has healthy mitochondria and has had its nucleus removed. The process can take place before or after the egg is fertilised using a man's sperm.

Although the genetic contribution of the egg donor is very small (1 per cent) and won't be detectable in the child's appearance and psychological characteristics, the transfer of genetic material affects the genetic constitution of the egg and the embryo. This means that changes will not only affect the child but also the child's descendants, and there has been criticism of the risk of introducing bad traits through the generations though there have been government assurances that the process will be closely monitored in the UK.

One of the distinctions that ought to play a crucial role in this debate is between genetic engineering that aims to remove a serious disability and engineering designed to make people more intelligent, better looking, stronger or more assertive. And it is the latter that has raised the spectre of eugenics.

Eugenics, of course, is reviled because of the policies adopted by a number of states in the first part of the 20th century, most famously the Nazis, to build a more productive and healthy population by eliminating from the gene pool those regarded it regarded as unfit. And the debate over the ethical implications of mitochondrial transfer is very much alive.

In the Council of Europe, 34 member politicians declared that the creation of babies from the DNA of three parents was a form of eugenics "incompatible with human dignity and international law". They claimed it contravened a European Union human rights convention that forbids genetic interventions that affect the human germ-line by altering the genome of descendants.

The thinking behind this prohibition is that tampering of this kind is not only dangerous, but makes humans into a product of engineering. Even if intentions are good, the use of such techniques undermines the reasons we have for respecting human individuals. Humans are supposed to be valuable in themselves. Products are merely means to ends.

The bad history of eugenics is a good reason why a state should not be allowed to use genetic technology for its purposes. But the practice of mitochondrial transfer and the motivation behind it have nothing to do with eugenics as it was once practiced. It would allow consenting parents to have a child who will not suffer from a serious disability.

Read more from the original source:
Three-person IVF has nothing to do with eugenics