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It’s an exciting time for gene therapy with a myriad of disease indications being explored ranging from blindness to potential cures for HIV and successful clinical trials being conducted for infants with Spinal Muscular Atrophy (SMA). These awesome advances have not been ignored by RSRT which is why we recently launched a Gene Therapy Consortium (GTC) that is undertaking key experiments to determine if this approach is a feasible strategy for Rett. Program Director, Tim Freeman had a chance to sit in on a GTC meeting in Boston recently and shared his perspective in this post.

Gene therapy in the traditional sense delivers healthy genes into the body by way of a vector (Trojan horse) to compensate for mutated genes. But what if you could repair a gene by splicing out the mutation with “molecular scissors” and replacing it with the correct bits of DNA? Genome editing, as it’s called, is sounding less like futuristic science fiction and more like a tangible treatment.

A revolutionary new technology, Crispr-Cas9, which capitalizes on a naturally occurring molecular phenomenon allows for the mutated bits of DNA to be snipped out and the correct bits to be inserted. While this technology is not yet ready for prime-time there is lots of research taking place and progress is quick-paced.

What if you could go right to the root cause of that disease and repair the broken gene? That’s what people are excited about,”

– Katrine Bosley, Editas Medicine

We encourage you to read this Wall Street Journal article to learn more about Crispr-Cas9.