Thursday, November 16, 2017

US scientists try 1st gene editing in the body

In recent years I have reported several times about CRISPR and gene editing. I am not a scientist, but, to a layman, this sounds like the best possibility for curing Kennedy's Disease. In the article referenced below, it discusses the potential benefits and possible dangers of gene editing.

There is much more research that needs to be done, but the possibility of a cure is now closer than ever before.

US scientists try 1st gene editing in the body


"...This time, the gene tinkering is happening in a precise way inside the body. It’s like sending a mini surgeon along to place the new gene in exactly the right location.

“We cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending,” said Dr. Sandy Macrae, president of Sangamo Therapeutics, the California company testing this for two metabolic diseases and hemophilia. “It becomes part of your DNA and is there for the rest of your life.”

That also means there’s no going back, no way to erase any mistakes the editing might cause.

“You’re really toying with Mother Nature” and the risks can’t be fully known, but the studies should move forward because these are incurable diseases, said one independent expert, Dr. Eric Topol of the Scripps Translational Science Institute in San Diego.

Protections are in place to help ensure safety, and animal tests were very encouraging, said Dr. Howard Kaufman, a Boston scientist on the National Institutes of Health panel that approved the studies.

He said gene editing’s promise is too great to ignore. “So far there’s been no evidence that this is going to be dangerous,” he said. “Now is not the time to get scared.”..."

A short APNews animation video does a good job of explaining the process. 

(AP Animation/Marshall Ritzel)

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