Structure and Function of Newly Discovered CRISPR Immune System
Utah State University
In tandem papers in Nature, researchers report effects of Cas12a2
Genetic Engineering
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
NIH Researchers Home In on a New Cause of Stargardt Disease
National Institutes of Health
Study may help lead to gene therapy for rare inherited blinding disease
New CRISPR-Cas Approach Permits More Precise DNA Cleavage
Massachusetts General Hospital
So-called SpRYgests enable DNA cutting at practically any sequence for DNA editing
Discovery Broadens Scope of Use of CRISPR Gene Editing
Northwestern University
Combining two powerful therapeutic technologies may change the landscape of medical therapeutics
Researchers Discover Dozens of Genetic Defects Important for Immune Defense
University of Helsinki
Results suggest that targeted inhibition of certain NFKB signaling pathway components is an viable therapeutic approach
How CRISPR-Cas9 Contributed to Rare Disease Research in 2021
Brydie Thomas-Moore, PhD
Research from this past year highlights the potential of CRISPR-Cas9 technology in modeling and treating rare disease
A New Generation of Malaria Vaccines
Miriam Bergeret, MSc
Promising results from the first in-human trials of genetically engineered malaria vaccines
The Therapeutic Potential of Bioengineered Bacteria
Masha G. Savelieff, PhD
Genetically modified microbes designed to treat human disease have entered into preclinical and clinical development