New research reveals an essential step in scientists' quest to create targeted, RNA-based, more eco-friendly fungicides that protect food crops.
Gene therapy has traditionally been conceptualized as a one-time, curative treatment option; however, research shows that there may be a need for subsequent doses years after initial treatment. While adeno-associated viral (AAV) vectors are a core part of this powerful therapeutic approach, they present two key challenges, immunogenicity and durability, in gene therapy. Researchers showed the benefits of a new technology to overcome these challenges and ultimately unlock the potential of gene therapy.
Researchers have discovered a new gene-editing technique that allows for the programming of sequential cuts -- or edits -- over time.
Huntington's disease is caused by a mutation in the Huntingtin gene (HTT), which appears in adults and features motor, cognitive and psychiatric alterations. The origin of this disease has been associated with the anomalous functioning of the mutated protein: mHTT, but recent data showed the involvement of other molecular mechanisms.
The remarkable genetic scissors called CRISPR/Cas9, the discovery that won the 2020 Nobel Prize in Chemistry, sometimes cut in places that they are not designed to target.
Runt-related transcription factor 1 (RUNX1) has been linked to retinal neovascularization and the development of abnormal blood vessels, which result in vision loss in diabetic retinopathy. Now, scientists have found that RUNX1 inhibition presents a new therapeutic approach in the treatment of age-related macular degeneration (AMD), which is the leading cause of blindness in the elderly worldwide. Their results are reported in The American Journal of Pathology, published by Elsevier.
Scientists have identified a way to "rescue" muscle cells that have genetically mutated, paving the way to a possible new treatment for rare childhood illness such as Duchenne Muscular Dystrophy (DMD).
Researchers from Indiana University have identified key genetic changes in the interstitial kidney tissue of people with diabetes, a discovery that signifies the potential for a revolutionary new genetic approach to the treatment of kidney disease. They will contribute their findings to the Kidney Precision Medicine Project's (KPMP) "cell atlas," a set of maps used to classify and locate different cell types and structures within the kidney.
MUSC Hollings Cancer Center researcher Yongxia Wu, Ph.D., identified a new target molecule in the fight against graft-versus-host disease.
A new study initiated by Wyss Core Faculty member George Church's Synthetic Biology team at Harvard's Wyss Institute for Biologically Inspired Engineering, and driven by a collaboration with Google Research has applied a computational deep learning approach to design highly diverse capsid variants from the AAV2 serotype across DNA sequences encoding a key protein segment that plays a role in immune-recognition as well as infection of target tissues. The approach has broad implications for designing more effective and safer gene therapies.