CRISPR Beyond Gene-Edited Babies

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In late November, it was hard to miss the shocking claim that a Chinese researcher used CRISPR, a relatively new gene-editing technique, on twin baby girls while in the embryonic state. According to the Associated Press, the researcher, He Jiankui, said his goal “was not to cure or prevent an inherited disease, but to try to bestow a trait that few people naturally have—an ability to resist possible future infection with HIV, the virus that causes AIDS.”

The procedure was quickly denounced by many scientists as unsafe and unethical, with the potential to harm not only the girls’ genes, but also their offspring. Many articles on the subject followed, debating the ethics and safety of the work. Adding to the controversy is that the research has yet to be verified in a peer-reviewed journal.

But how did we get here? What is CRISPR and its intended use? For people who want a little more background on this groundbreaking technique, and its importance beyond the latest headlines, we invite you read our new briefing paper on CRISPR and its use in neuroscience—both as a research tool and, potentially, a treatment for brain-related disorders.

This technique allows you to easily make permanent genetic changes at the site being targeted, says Sam Sternberg, Ph.D., an assistant professor in the department of biochemistry and molecular biophysics at Columbia University…. For the development of human therapeutics, scientists can use CRISPR to repair a gene with a mutation that is associated with disease. Alternatively, CRISPR can be harnessed to introduce site-specific mutations, enabling the study of how diseases progress in the first place.

The paper also delves into a number of the current challenges and limitations, which help to shed light on concerns over gene-editing in humans at this stage. Sternberg shared:

In the context of a three billion letter genome, there is the possibility that Cas9 might accidentally target and cut the wrong sequence, he says. This is a big concern as the changes you make with CRISPR are permanent—and you don’t want to introduce mutations to other places in the genome and cause lasting effects that you never intended to cause.

Hear more from Sternberg and other top experts in the CRISPR field in our paper, “CRISPR and the Brain.”

–Ann L. Whitman

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