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The CRISPR-Cas9 Cancer Therapy

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News: Latest – distributed around Yorkshire June 24, 2016 11:38:30 AM

A team of Physicians at the University of Pennsylvania’s School of Medicine now has their project of modifying the immune cells of 18 different cancer patients with the CRISPR-Cas9 system approved by the National Institute of Health.

Targeted genome editing using engineered nucleases has rapidly gone from being a niche technology to a mainstream method used by many biological researchers.

This widespread adoption has been largely fueled by the emergence of the clustered, regularly interspaced, short palindromic repeat (CRISPR) technology, an important new approach for generating RNA-guided nucleases, such as Cas9, with customizable specificities.

Genome editing mediated by these nucleases has been used to rapidly, easily and efficiently modify endogenous genes in a wide variety of biomedically important cell types and in organisms that have traditionally been challenging to manipulate genetically.

Furthermore, a modified version of the CRISPR-Cas9 system has been developed to recruit heterologous domains that can regulate endogenous gene expression or label specific genomic loci in living cells. Although the genome-wide specificities of CRISPR-Cas9 systems remain to be fully defined, the power of these systems to perform targeted, highly efficient alterations of genome sequence and gene expression will undoubtedly transform biological research and spur the development of novel molecular therapeutics for human disease.

CRISPR is the gift that keeps on giving—when it’s not fighting blindness, tackling HIV, or even recording real-time immune responses, it is taking on the emperor of all maladies: cancer.

But what’s even more fascinating about this use of CRISPR is that the National Institute of Health’s (NIH) Recombinant DNA Research Advisory Committee (RAC) has approved the first-ever use of CRISPR in human cancer therapy, a monumental step in the history of the gene-editing technology.

While receiving funding from the Parker Immunotherapy Foundation, physicians at the University of Pennsylvania School of Medicine plan on working with the T-cells of 18 patients who have either melanoma, sarcoma, or myeloma by performing three distinct CRISPR edits:

Offensive: T-cells will be modified to have a gene that produces a protein that will help T-cells identify and target cancer cells.

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