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Aav Vectors

All articles tagged with #aav vectors

health2 years ago

Revolutionary Gene Therapy Treats Age-Related and Genetic Hearing Loss

Researchers at Mass Eye and Ear have successfully used adeno-associated virus vectors in gene therapies to treat genetic hearing loss in aged mouse models, pointing to the potential of virally mediated gene therapies for treating genetic hearing loss in the aged population. By 2050, one in 10 individuals is expected to live with some form of hearing loss, and genetic hearing loss is often the most difficult to treat. The breakthrough suggests that a virally mediated gene therapy could potentially treat genetic hearing loss, either by itself or in combination with a cochlear implant.

health2 years ago

Gene Therapy Offers Hope for Age-Related Hearing Loss

Researchers have successfully reversed age-related genetic hearing loss in aged animal models using adeno-associated virus (AAV) vectors carrying a healthy TMPRSS3 gene. This breakthrough suggests the potential of gene therapies to treat genetic hearing loss, even in advanced age. The study demonstrates for the first time the effectiveness of AAV vector-mediated gene therapy in reversing hearing loss in aged mice, opening possibilities for similar treatment in humans.

science2 years ago

Prime Editing Successfully Alters Genes in Live Mice.

Harvard researchers have developed an optimized prime editing method that can alter genes in living mice, marking a major advance in gene editing for studying and treating genetic disorders. The researchers identified bottlenecks limiting the effectiveness of adeno-associated virus (AAV)-mediated prime editing in vivo and developed vectors with increased prime editing expression, RNA stability, and modulation of DNA repair. The resulting prototype for optimized prime editing, v3em PE-AAVs, achieved therapeutically relevant levels of prime editing in the mouse brain, liver, and heart with no detected off-target editing.

health2 years ago

Revolutionizing Gene Therapy for Neurodegenerative Diseases with Enzyme Discovery.

Researchers have successfully delivered adeno-associated virus (AAV) vectors into specific brain regions involved in Parkinson's disease by creating a successful blood-brain barrier opening using low-intensity focus ultrasound guided by magnetic resonance imaging. The less-invasive nature of this methodology could facilitate viral vector delivery for gene therapy, making theoretical interventions to treat neurodegenerative disorders a reality.