CRISPR Clinical Trial to Edit Human Genes

World’s first gene editing technology, CRISPR-Cas9, which uses CRISPR to tackle many deadly diseases like Cancer, is collectively affirmed on 21st June by a review panel at National Institutes of Health (NIH). This trial is specially designed to treat three kinds of cancer. The first trial aims to test whether it is safe for people and is expected to encourage many other medical groups to initiate the use of CRISPR in treatments.

The trail which is headed to start later this year is surprisingly funded by the tech billionaire Sean Parker (Former President at Facebook & Napster Founder). The aim of the Parker Institute of Cancer Immunotherapy’s revolutionary project is to solve cancer. The research led by Dr. Carl June is backed by $250 million, 300 scientists and 40 labs.

On Tuesday, Penn’s Dr. Carl June told the National Institute of Health’s Recombinant DNA Advisory Committee (RAC), “Our preliminary data suggests that we could improve the efficacy of these T cells if we use CRISPR.”

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Scientists at the University of Pennsylvania (UPenn) who proposed the experiment claimed that the technique used will remove certain cells from the immune system called T-cells from the cancer patients. The trail experiment would also be conducted at MD Anderson Cancer Center in Texas (9 patients) and the University of California, San Francisco (3 patients). Penn shall also produce genetically modifies T cells.

After genetically modifying the T cells with the use of CRIPSR (Clustered regularly interspaced short palindromic repeats), they are reprogrammed back into the patient body which targets and devastate the tumor cells like myeloma, melanoma, and sarcoma. Under this gene editing an entirely new gene is added to these T-cells for a receptor that bonds to a protein called NY‐ES0‐1.

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Well, the experiment is still not approved by the Food and Drug Administration (FDA), which regulates clinical trials. Affirmation of the regulators at the institutions, where the project will be worked is also required.

Even though the studies have found bright results with genetically modified T’s, unfortunately the cells have been shown to work only on few types of blood cancers like leukemias. Results were disappointing with solid tumors. So, there are possible chances for patients to come back with cancer as the tumor repels the T-cells.

Earlier this month, Dr. June in Forbes Philanthropy Summit said that the aim of the experiments is to produce T cells, a type of white blood cell, “better than nature made them.” The cells will be edited to lack several genes, including one that allows them to respond to a protein called PD-1.

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The gene editing has already been used in humans earlier, but just not done using the CRISPR-Cas9 system. TALEN was the first genome editing technique which was used last year to modify T-cells to treat a British toddler suffering with leukemia.

Editas Medicine, a biotech company which is based in Cambridge, Mass., is expected to be the first to use CRISPR in people to treat a rare eye disease. But Penn is expected to defeat Editas. Seattle’s Juno Therapeutics promised Editas, almost $737 million for CRISPR technology, while Novartis is at a $280 million with Intellia Therapeutics.

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