CRISPR for Somatic Therapy


By Dr. Kevin Lee


A powerful new genetic technique with the unwieldy name of CRISPR/Cas9, or just CRISPR (pronounced like “crisper”) has attracted monumental media coverage, yielded major prizes for its co-discoverers, and spawned intense patent disputes. The method, which has been likened to “molecular scissors”, enables the precise editing of genes in virtually any organism, including humans. As a consequence, this technology has enormous potential for driving biomedical discovery and for treating human disease. At the same time, there has been significant (and well founded) concern about the possibility that the technique might be used to alter genes in human embryos and even allow the transmission of altered genes across generations.

There is good reason to be concerned about some potential uses of gene editing technologies – specifically when they involve non-therapeutic purposes, or are employed to alter the genomes of future generations. A recent “Gene Editing Summit” of the National Academies tackled these thorny issues. And of course, safety is a primary concern – this technique must be thoroughly and rigorously evaluated to ensure that genetic mistakes are not created in the process of using this method. But what is sometimes overlooked in this discussion is that certain applications of this genetic technology fall well within the boundaries of well established, carefully regulated gene therapies. Most diseases play out in the body’s somatic cells, not in “germline” cells from which genetic information is passed on to subsequent generations. Gene editing-based treatments in somatic cells for life-threatening illnesses is an area of tremendous medical need, and is not subject to the same ethical concerns as genetic manipulation of human embryos. However, up to now, the perspectives of the patients and families that would first benefit from these ethical somatic gene editing technologies has received little attention.

“As a parent with an incredibly sick child, what are we supposed to do — sit by on the sidelines while my child dies? There’s zero chance of that,” Wilsey says. “CRISPR is a bullet train that has left the station — there’s no stopping it, so how can we harness it for good?”

In a current Nature News article, Matt Wilsey comments on these issues and expresses his hope that concerns about gene editing in human embryos don’t overshadow legitimate uses of the technology to benefit patients suffering from life threatening genetic disorders.

These are important discussions – safety, ethics, and access – but at the same time, we have a moral obligation to deliver safe, rigorously tested life-saving therapies to alleviate the suffering of patients with serious illnesses. In addition, we have and will continue to use gene editing techniques like CRISPR as a laboratory tool to understand genetic diseases, to create cellular and animal models of diseases, and test candidate therapies. Patient voices must be heard: Lives depend on it.

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