This is a follow on to a previous post – Is this the Person of the Century? The TERRIFYING Implications of CRISPR/Cas9.
From New York Times online 31 December 2015:
Duchenne muscular dystrophy is a progressive muscle-wasting disease that affects boys, putting them in wheelchairs by age 10, followed by an early death from heart failure or breathing difficulties. The disease is caused by defects in a gene that encodes a protein called dystrophin, which is essential for proper muscle function.
…gene therapy — the idea of curing a genetic disease by inserting the correct gene into damaged cells — is making a comeback. A new technique, known as Crispr-Cas9, lets researchers cut the DNA of chromosomes at selected sites to remove or insert segments.
Three research groups, working independently of one another, reported in the journal Science on Thursday that they had used the Crispr-Cas9 technique to treat mice with a defective dystrophin gene. Each group loaded the DNA-cutting system onto a virus that infected the mice’s muscle cells, and excised from the gene a defective stretch of DNA known as an exon.
BOTTOM LINE: Three groups of medical researchers succeeded in repairing defective genes in muscle cells. At least in mice it worked. The three are The Gersbach Lab at Duke University led by Charles A. Gersbach, The Olson Laboratory at University of Texas Southwestern Medical Center and the Wagers Laboratory, leader Amy J. Wagers, at Harvard University.
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