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For the first time, scientists were able to correct the genetic mutation that causes sickle cell disease in stem cells.
In a collaborative effort, researchers at UC Berkeley, UC San Francisco Benioff Children's Hospital Oakland Research Institute (CHORI), and the University of Utah School of Medicine fixed the mutation in modified stem cells from patients with the condition using a CRISPR/Cas9 gene editing approach.
The study, "Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells," was published in the journal Science Translational Medicine.
The scientists hope to re-infuse patients with the modified stem cells and alleviate disease symptoms.
"We're very excited about the promise of this technology," Jacob Corn, senior author on the study and scientific director of the Innovative Genomics Initiative at UC Berkeley, said in a news release. "There is still a lot of work to be done before this approach might be used in the clinic, but we're hopeful that it will pave the way for new kinds of treatment for patients with sickle cell disease."
(Score: 2) by Magic Oddball on Sunday October 23 2016, @03:37AM
One of my hospital roommates in 1990 was a toddler/preschooler who was very sick and in a LOT of pain thanks to SCD anemia. Out of the 20+ kids I shared rooms with over the years, she was one of the very few who made no effort to interact or play, and I don't think they expected her to reach puberty.
Obviously treatments for SCD are already a hell of a lot better than back then, but it's still a pretty nasty disorder for someone to be saddled with, so I'm glad they've found a likely solution.