Rajvinder Karda

This study compares AAV9 and AAV-F capsids for treating PDHD in mice, finding AAV-F improves survival and enzyme activity at lower doses but causes motor impairments.

This paper demonstrates the use of AAV9 vectors to deliver AntagoNATs targeting a natural antisense transcript in a DS mouse model, improving survival and reducing seizures.

This study demonstrates that AAV-delivered RNA editing machinery improves survival and reduces seizures in a mouse model of Dravet Syndrome.

This study shows that ascending vaginal E. coli infection in pregnant mice causes preterm birth and neonatal morbidity, with validated therapeutic potential for cervical gene therapy.

The paper identifies manufacturing plasmid sequences and disrupted vector genomes in liver tissue from a patient with AAV gene therapy-related hepatitis.

This study demonstrates that AAV9-mediated gene therapy prevents liver dysfunction in a mouse model of DGUOK deficiency.

The paper develops TDP-REG, a system using cryptic splicing to enable precise gene therapy activation in diseased neurons for ALS/FTD.

This study investigates spinal cord pathology in a Dravet Syndrome mouse model and identifies dysregulated pathways related to monooxygenase activity, fatty acid ligases, and lactate transporters.

The paper develops a method using adeno-associated virus vectors to generate somatic-transgenic mice for non-invasive monitoring of signaling pathways in disease models.

This paper demonstrates that gene therapy using AAV8 vector corrects urea cycle and citrulline-NO cycle defects in ASL-deficient mice, reducing neuronal disease and improving behavior.