Researchers Beginning To Use CRISPR In Studies

August 26, 2019

By Bio-IT World Staff

August 26, 2019 | After years of discussion, patent lawsuits, and hype, the gene-editing tool know as CRISPR is finally making its way into clinical trials.

NPR recently reported researchers at the Sarah Cannon Research Institute in Nashville, Tennessee using the gene-editing technique to treat patients suffering from sickle cell disease.

The treatment is part of a larger study being conducted by Vertex alongside CRISPR Therapeutics.

According to CNN, the study aims to recruit up to 45 adults with severe sickle cell disease.

"In the CRISPR trial, doctors aim to increase the production of a different kind of hemoglobin: fetal hemoglobin, which makes it harder for cells to sickle and stick together," CNN's Michael Nedelman and Minali Nigam write. "This process involves removing premature cells from the bone marrow and modifying them—using CRISPR in the lab—to eventually produce fetal hemoglobin. After a round of chemo, these cells are given back to the patient, replacing cells that were there."

It will likely take several months to determine whether the treatment yields any fruit, and NPR's Rob Stein reports it may take years before researchers can determine the longevity of related benefits.

This isn't the only research project utilizing the gene-editing technology. Researchers at the University of California, San Diego (UCSD) are using CRISPR to take "a step toward a gene therapy that could block severe pain caused by diabetes, cancer, or car accidents without the addictive effects of opioids," Antonio Regalado reports in MIT Technology Review.

For this project, UCSD researchers injected CRISPR-modified viral particles into the cerebral spinal fluid of mice in the hopes of temporarily blocking a key, pain-transmitting neuron.

Again, while initial results are promising, early findings have left plenty of unanswered questions.