3 ms·

Back in 2017, 60 minutes covered a group that was cured of sickle cell disease with gene therapy. How does this approach differ when they already showed success

• el_cujo 7y ago
  Just skimming through the article you posted, it says trials with a similar treatment resulted in one patient dying from a massive immune response and two other got cancer. Sounds like it relies on a virus to deliver the DNA, but I didn't read too carefully, so maybe CRISPR will turn out to have fewer side effects or a greater success rate. I'm pretty sure one of the up-sides to CRISPR is compared to other gene-editing techniques it's relatively cheap, which is always a plus.
  • maxerickson 7y ago
    The death and cancers mentioned over there were from gene therapy trials in general, not the specific study in the link.
• maxerickson 7y ago
  This study uses a different vector and targets a different gene. The NIH study uses a modified HIV to introduce the complete gene for the defective protein. https://www.nhlbi.nih.gov/news/2019/sickle-cell-patients-recovery-after-gene-therapy-heightens-hopes-cure https://www.nhlbi.nih.gov/news/2019/sickle-cell-patients-rec... This study uses CRISPR/Cas9 to increase expression of a gene that the patients already have.