07Sep 2023
CRISPR-Cas9 Used to Edit HBG1 and HBG2 Promoters in Groundbreaking Sickle Cell Disease Treatment

CRISPR-Cas9 Used to Edit HBG1 and HBG2 Promoters in Groundbreaking Sickle Cell Disease Treatment

A study has made significant strides in treating sickle cell disease using CRISPR-Cas9 technology to elevate fetal hemoglobin levels in red blood cells. In a phase, 1-2 clinical study, autologous OTQ923, edited with CRISPR-Cas9 and gRNA-68, demonstrated sustained induction of fetal hemoglobin (19.0% to 26.8% of total hemoglobin) in three severe sickle cell disease patients. This innovative approach showed clinical improvements and decreased disease manifestations.

  • #critical care

Like

Save

Share