gene therapy

A new method called “REVeRT” expands the capabilities of gene delivery platforms by transporting larger genes than previously possible.  

Biologics like monoclonal antibodies and mRNA vaccines are complex drugs. Yongchao Su uses biophysical tools and innovative strategies to understand them better.

Researchers created an improved method for assays that detect viral vectors in a patient’s blood or urine. It’s used to ensure that bystanders will not be exposed.

Researchers discovered an immunological connection between the eye and the brain that could lead to new therapeutics for central nervous system diseases.

People with achromatopsia have never seen color. Restorative gene therapies have had mixed success, leaving researchers wondering why.

The durability and capacity to boost protein expression of circular RNA makes it a promising candidate for next-generation vaccines and gene therapies.

A gene therapy targeting excitatory neurons significantly reduced seizures in a mouse model of the most common cause of child epilepsy.

Using a gene therapy approach, researchers relieved pain behaviors in mice and tested the approach in human cells.

Experts discussed the major hurdles of AAV gene therapy, including immunogenicity, vector design, and AAV variability.

Researchers identified a peptide that selectively blocks pain signaling in sensory neurons and packaged it into a viral-based gene therapy in a step towards treating chronic pain without the risk for addiction.