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NEW GENE THERAPY: 'When I saw it working in my own lab, my jaw dropped,' Nobel winner scientist says

By Catholic Online (NEWS CONSORTIUM)
November 8th, 2013
Catholic Online (www.catholic.org)

Entitled "Crispr," a new form of DNA modification is astounding the medical and scientific communities. The new technique could make serious inroads on incurable diseases, and halt conditions in unborn fetuses.

LOS ANGELES, CA (Catholic Online) - "Crispr" comes with a very high commendation: Nobel Prize winner Professor Craig Mello described the new technique as "a real game-changer . Crispr is absolutely huge," Mello writing in the Independent said. "It's incredibly powerful and it has many applications, from agriculture to potential gene therapy in humans.

"This is really a triumph of basic science. It's a tremendous breakthrough with huge implications for molecular genetics. It's a real game-changer.

"It's one of those things that you have to see to believe. I read the scientific papers like everyone else but when I saw it working in my own lab, my jaw dropped. A total novice in my lab got it to work," Mello says.

Crispr "lowers the threshold" for carrying out gene therapy on human IVF embryos, Mello adds.

For the first time, Crispr will allow scientists to edit genetic information with great precision. It will potentially allow scientists to treat genetic disorders such as sickle-cell anemia, Down syndrome and Huntington's disease.

Some believe that the technique is so accurate it might be used for gene therapy, replacing faulty genes with healthy ones on incurable viruses, such as HIV and cancer.

Also - in what could have controversial and ethical reverberations - Crispr might also be used to correct gene defects in human IVF embryos, allowing disorders to be removed before a baby is born.

Gene therapy has previously relied upon using viruses to insert DNA at random into the human genome, which is an inaccurate and risky process.

Professor Mello said the new technique could address many of the safety concerns of "germline" gene therapy because it is so accurate, although he warned that its use in IVF was some way off because potential "off-target effects' could lead to 'unintended consequences."

Dr. Dagan Wells, an IVF scientist at Oxford University, is excited with the new developments. "If this new technique succeeds in allowing perfectly targeted correction of abnormal genes, eliminating safety concerns, then the exciting prospect is that treatments could be developed and applied to the germline, ridding families and all their descendants of devastating inherited disorders.

"It would be difficult to argue against using it if it can be shown to be as safe, reliable and effective as it appears to be. Who would condemn a child to terrible suffering and perhaps an early death when a therapy exists, capable of repairing the problem?'

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