Terence R. Flotte

This study reports 5-year safety and 3-year clinical outcomes of AAV9-GLB1 gene therapy in a juvenile GM1 gangliosidosis patient, showing sustained seizure freedom and biochemical improvements.

This phase 1/2 trial evaluates AAV9 gene therapy for GM1 gangliosidosis Type II, showing safety and some biochemical and neuroimaging improvements.

The paper identifies an amino acid variant in AAV capsid VR-1 that reduces liver targeting, suggesting its potential for minimizing liver toxicity in gene therapy.

This paper demonstrates that a compact ABE corrects the PiZ mutation in AATD mouse models, reducing liver disease and improving lung function.

This study characterizes immune responses to AAVrh8 gene therapy in CNS patients and evaluates the effectiveness of immunosuppression.