@Kohn_Lab
The FDA has granted accelerated approval for KRESLADI, a gene therapy for LAD-I — a rare immune disorder that can be fatal in young children.
Led by @Kohn_Lab @UCLA, all 9 clinical trial patients survived with restored immune function.
🔗bit.ly/4uVBcfg #GeneTherapyView quoted post on X
www.nejm.org Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency | NEJM Severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency (ADA-SCID) is a life-threatening inborn error of immunity for which lentiviral gene therapy has been investigated... A blood stem cell gene therapy co-developed by @UCLA's Dr. Donald Kohn restored immune function in 59 of 62 children with ADA-SCID, a rare and fatal immune disorder, with no serious complications reported. #ResearchPowersProgress @Kohn_LabView quoted post on X
stemcell.ucla.edu/news/gene-ther…
A new study in @NEJM reports that an investigational gene therapy restored immune function in 9 children with LAD-I, a rare & life-threatening immune disorder. Those children are now living symptom-free 2 years after treatment in clinical trials co-led by @Kohn_Lab @UCLA.View quoted post on X
