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Gene Therapy

The First Human CRISPR Trials

Posted on 20 June 2016

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A cancer treatment involving CRISPR technology has been proposed to a federal safety board

CRISPR technology is less than 5 years old, but it’s heralded an optimistic boom in the genome editing field, with increasing attempts to translate the ground-breaking tech to clinical advances. Editas Medicine, a company set up by a group of CRISPR’s founders, has already claimed it aims to conduct a trial in 2017 for Leber congenital amaurosis, a rare form of blindness. 

Now, a group from the University of Pennsylvania is proposing a treatment to target myeloma, melanoma, and sarcomas; editing blood cells outside the body and injecting them back into the body newly corrected. The University already specialises in a similar treatment, involving genetically modifying a patient’s immune cells to attack cancer cells. 

Researchers in the field of gene transfer are excited by the potential of utilizing CRISPR/Cas9 to repair or delete mutations that are involved in numerous human diseases in less time and at a lower cost than earlier gene editing systems”

Similar editing treatments have actually been conducted already, for HIV and leukemia, but these did not use CRISPR which is much easier, faster and cheaper in comparison to other editing technologies like TALENs or ZFNs (zing finger nucleases). The Recombinant DNA Advisory Committee is now reviewing their proposal, which involves a removal of 2 genes: one of these being PD-1 which is an inhibitor of the body’s immune response cancer frequently uses to evade attack. These treatments can be risky, but they have great potential in the field of cancer therapy. If the proposal goes to plan and CRISPR proves both successful and safe in upcoming trials, it will bode well for an expansion into longevity based treatments. 

Read more at Technology Review

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