Structure and Function of Newly Discovered CRISPR Immune System
Utah State University
In tandem papers in Nature, researchers report effects of Cas12a2
gene therapy
Tips for Transitioning Cell & Gene Therapies to the Clinic
Dominic Clarke, PhD
How to circumvent common barriers to bringing cell and gene therapies to the clinic
FDA Approves First Gene Therapy to Treat Adults with Hemophilia B
U.S. Food and Drug Administration
The one-time gene therapy, Hemgenix, was evaluated in two studies and was found to decrease annualized bleeding rate
Removing Roadblocks in Cell and Gene Therapy Quality Control
Marwan Alsarraj, MS
Droplet digital PCR (ddPCR) technology provides unmatched quality control for cell and gene therapies
FDA-Approved Gene Therapy for Transfusion-Dependent Beta-Thalassemia Offers Potential Cure
María Carla Rosales Gerpe, MSc, PhD
Patients with transfusion-dependent beta-thal will soon be treated with lentiviral vector gene therapy Zynteglo to restore hemoglobin levels
A Look Ahead: Clinical Diagnostics and Cell & Gene Therapies
Marwan Alsarraj, MS
The pandemic has fueled new market growth and will continue to shape the future of diagnostics
COVID-19 Is Spurring Innovation in Cell & Gene Therapy Development
Rupa Pike, PhD
While the pandemic slowed CGT development, it accelerated potential regulatory pathways for new therapies
Gene Therapy Restores Immune Function
Miriam Bergeret, MSc
Researchers have successfully treated a rare genetic immunodeficiency disorder using an investigational gene therapy
Scientists Discover a New Genetic Form of ALS in Children
National Institute of Neurological Disorders and Stroke
Study links ALS to a fat manufacturing gene and maps out a genetic therapy
Gene Therapy Shows Promise in First Trial for Patients with Childhood Blindness
University of Pennsylvania School of Medicine
The gene therapy was safe and improved patients' vision, according to initial data from a clinical trial