Sunday, October 18, 2015

Re-editing the human genome for disease therapy

Researchers at Washington State decided that they could figure out a new way to edit genes.  The typical gene-editing protocol started with using nucleases to induce double-stranded DNA breaks then allowing the cell’s non-homologous end-joining machinery to repair the nucleic acid.  This procedure didn’t always work correctly.  The researchers learned that if they tapped into the cell’s recombination repair pathway was a more efficient and precise way to edit the gene.  The new method can be used to disrupt a gene and could also insert new information into a specific site in the genome.  The Washington State Researchers used the new method to disrupt and replace CCR5 in the human T cells.   They also delivered a repair template that coded for gene they wanted to knock in. That gene was flanked by sequences that match the target gene.  The researcher demoed the success of their method with a reporter gene and then with gene cassettes with therapeutic abilities such as rewriting the CCR5 gene to protect T cells from HIV infections.  The study could be used to combat and correct other diseases like cancer. 
     This could be great for people who have diseases that doesn't have a cure for yet.  My wife have a disease that doesn't have a cure just yet, Reflex Sympathetic Dystrophy  RSD.  The only treatment allowed in the US is pain medication or anti-seizure.  I know there is a company in PA that is working on non-narcotic pain medication but maybe one day this could be used to repair the pain receptors or the affected cell to return to normal function. 

The original article
Genome editing cuts out HIV

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