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March 4, 2020 06:40 pm
Original Link: http://rss.slashdot.org/~r/Slashdot/slashdot/~3/B9Ig0IxIzBk/crispr-used-to-edit-genes-inside-a-patient-with-a-rare-form-of-blindness
CRISPR Used To Edit Genes Inside a Patient With a Rare Form of Blindness
An anonymous reader quotes a report from NPR: For the first time, scientists have used the gene-editing technique CRISPR to try to edit a gene while the DNA is still inside a person's body. The groundbreaking procedure involved injecting the microscopic gene-editing tool into the eye of a patient blinded by a rare genetic disorder, in hopes of enabling the volunteer to see. They hope to know within weeks whether the approach is working and, if so, to know within two or three months how much vision will be restored. Before this step, doctors had only used CRISPR to try to treat a small number of patients who have cancer, or the rare blood disorders sickle cell anemia or beta-thalassemia. In this new experiment, doctors at the Casey Eye Institute in Portland, Ore., injected (into the eye of a patient who is nearly blind from a condition called Leber congenital amaurosis) microscopic droplets carrying a harmless virus that had been engineered to deliver the instructions to manufacture the CRISPR gene-editing machinery. Beginning in infancy, the rare genetic condition progressively destroys light-sensing cells in the retina that are necessary for vision. Vision impairment with LCA varies widely, but most patients are legally blind and are only able to differentiate between light and dark or perhaps to detect movement. The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the genetic defect that caused the blindness. That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells -- enabling patients to regain at least some vision.Read more of this story at Slashdot.
Original Link: http://rss.slashdot.org/~r/Slashdot/slashdot/~3/B9Ig0IxIzBk/crispr-used-to-edit-genes-inside-a-patient-with-a-rare-form-of-blindness
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