Ahad A. Rahim

This paper develops a pipeline to identify and evaluate novel manganese chelators using zebrafish and mouse models, finding that H3PyC3A and Me3PyC3A show improved manganese mobilization and reduced accumulation.

This study demonstrates that CRISPR-CasRx reduces C9orf72 repeat RNAs and DPRs in vitro and in vivo, suggesting its therapeutic potential for ALS/FTD.

This study investigates the impact of LRRK2 G2019S mutation on Wnt and NFAT signaling in Parkinson's disease models, finding significant dysregulation in specific brain regions.

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 study shows that neonatal brain-directed gene therapy using AAV9-CLN6 significantly improves survival, motor function, and reduces neuropathology in a mouse model of CLN6 Batten disease.

This paper develops a non-integrating lentiviral gene therapy for refractory focal epilepsy, showing reduced seizure frequency in a rat model.

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.