The clinical trial that Mapara enrolled his patient in last year was one of the first to attempt to use CRISPR to treat a genetic disorder in humans. Potential for good - clinical trials show promise Sickle cell disease and beta thalassemia Still, the technology comes with significant ethical implications, including ensuring it does not have unintended negative consequences, that it is used equitably, and that a consensus is reached on where to draw the line in the technology’s use.ĪAMCNews spoke with researchers, physicians, ethicists, and educators on the cutting edge of CRISPR technology about its enormous potential for both good and harm to the future of humanity. CRISPR has been used to experiment with gene-edited mosquitos to reduce the spread of malaria, for engineering agriculture to withstand climate change, and in human clinical trials to treat a range of diseases, from cancer to transthyretin amyloidosis, a rare protein disorder that devastates nerves and organs. The technology has been celebrated throughout the scientific community as a significant advancement that is changing the way research is done across fields. “It’s a tool that scientists and clinicians around the world are using to understand our genetics, the genetics of all living things, and - most importantly - to intervene in genetic disease,” Doudna said during a speech at Learn Serve Lead 2021: The Virtual Experience, the AAMC’s annual meeting, in November. ![]() The enzyme then works like a pair of scissors, cutting the DNA’s double helix and allowing for sequences to be deleted, added, or replaced. In particular, Doudna and Charpentier found that an enzyme known as Cas9 can be guided by a programmable RNA to locate specific genetic sequences in any organism. The scientists worked together to uncover precisely how bacteria have evolved to fight off viruses - and to apply that same process to engineer human cells. Jennifer Doudna, PhD, University of California, BerkeleyĬRISPR, as it is known today, was developed by two scientists, Jennifer Doudna, PhD, who runs a lab at the University of California, Berkeley, and Emmanuelle Charpentier, PhD, scientific and managing director of the Max Planck Unit for the Science of Pathogens in Berlin, Germany, who were awarded the 2020 Nobel Prize in chemistry for their work on this technology. “ a tool that scientists and clinicians around the world are using to understand our genetics, the genetics of all living things, and - most importantly - to intervene in genetic disease.” “ may reject them, or the donor cells might attack the recipient.”īut with the development of CRISPR (which stands for clustered regularly interspaced short palindromic repeats), new opportunities for treatment using the patients’ own cells have opened up. ![]() “You’re running into risks of introducing foreign cells into a recipient,” Mapara says. “It has a huge impact on their quality of life and well-being.”Īt the moment, a bone marrow transplant from a healthy donor is the only curative option, but this approach can have severe complications. ![]() “This is a huge health problem for these patients,” Mapara says. The abnormal cells take on a sickle, or curved, shape, which can clot within narrow blood vessels. Sickle cell disease, which currently affects about 100,000 people living in the United States and millions worldwide, is the result of a genetic mutation that produces an abnormal type of hemoglobin, the protein that red blood cells use to deliver oxygen throughout the body. “ doing phenomenally well,” says Mapara, who is a hematologist, oncologist, and blood transplant physician. It has been more than a year since Markus Mapara, MD, a professor of medicine and director of blood and marrow transplantation at Columbia University Irving Medical Center in New York, first used an experimental CRISPR gene-editing treatment in a patient with sickle cell disease, an inherited blood disorder that can cause severe pain, organ damage, and premature death.Īlthough the clinical trial is still in early stages and has only been tested in a few patients, so far, the results are promising. CASGEVY is a treatment for sickle cell disease and was tested in a clinical trial discussed in this article. Food and Drug Administration (FDA) announced the approval of CASGEVY, the first FDA-approved therapy that uses CRISPR/Cas9 gene-editing technology.
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