New Orleans—In the wake of the highly publicized and tragic death of a patient who participated in a gene therapy trial at the University of Pennsylvania comes some good news: encouraging early results from two studies reported here at the annual meeting of the American Society of Hematology, one aimed at a lethal form of inherited immunodeficiency and the other targeting hemophilia B.
If the promising but preliminary results from the two phase 1 trials hold up, the two studies may be the first to provide evidence of clinical benefit for patients treated with gene therapy.
Stephenson J. Gene Therapy Trials Show Clinical Efficacy. JAMA. 2000;283(5):589–590. doi:10.1001/jama.283.5.589
Coronavirus Resource Center
Customize your JAMA Network experience by selecting one or more topics from the list below.