Successful CRISPR treatment of a Sickle Cell patient

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Encouraging news that the technology can potentially cure a number of genetic disorders

https://www.npr.org/sections/health...progress-in-fight-against-sickle-cell-disease

Frangoul and other researchers caution, however, that the results involve just one patient who was only recently treated. It is far too soon to answer the most crucial questions: Will the modified-cell treatment continue to improve the patient's health? Will the treatment keep working? Will it help her live longer? Is it safe in the long term?

"We are hoping it is" a success, Frangoul says. But "it is still too early to celebrate."

NPR has exclusive access to chronicle the experience of Frangoul's patient, Victoria Gray of Forest, Miss., the first person with a genetic disease to be treated with CRISPR in the United States.

"So look at this," Frangoul said recently with a smile, as he showed Gray her latest blood test results. The testing indicated that the genetically modified cells had already started producing the crucial protein at levels doctors hope will alleviate her suffering.

"I am super-excited about your results today," Frangoul said.In July, Gray was recovering from the medical procedure, which involved using an experimental technique called CRISPR to edit the genes of her own bone marrow cells.
Meredith Rizzo/NPR
While Gray knows it's still very early, she described how the treatment appears to be helping her. She has not suffered any of the painful attacks that torture sickle cell patients and has not needed to rush to the hospital for care since getting the modified cells this summer. She has not needed any blood transfusions either and has begun to reduce the pain medication she had been taking chronically.

"It's a miracle," says Gray, who says she has hope for the first time after a lifetime of struggling with excruciating pain and debilitating, life-threatening complications of the disease. Sickle cell disease is an inherited condition that is marked by defective oxygen-carrying red blood cells.

 

[Ygggdrasil]

The results aren't so surprising given reports from ~ 2 years ago showing that gene therapy could have similar effects on sickle cell anemia (https://www.physicsforums.com/threa...in-one-patient-for-at-least-15-months.906352/). In fact, the magnitude of the effect seems quite similar (e.g. both studies reports ~50% of the patients' hemoglobin has been corrected). However, the CRISPR approach would likely be the safer approach. The gene therapy approach involves using lentivirus to randomly insert a β-globin gene into hematopoietic stem cells, but this approach involves the risk of the inserted gene disrupting a tumor-suppressor gene in the stem cells, which would increase the risk that the engineered cells could become cancerous. In contrast, the CRISPR approach involves targeted editing at a specific site in the genome, which would carry much less risk of adverse effects as long as CRISPR does not induce anyhttps://www.physicsforums.com/threads/measuring-off-target-mutations-during-https://www.physicsforums.com/insights/dont-fear-crispr-new-gene-editing-technologies-wont-lead-designer-babies/-gene-editing.967151/ in the genome.

The report is definitely a good step forward towards a cure for sickle cell disease and towards the wider use of CRISPR in the clinic. Of course, the information reported by NPR is based only on one patient and has not yet been published in a peer-reviewed scientific journal, so we'll have to wait until the trial concludes to know definitively whether the CRISPR-based treatment is safe and effective.