Özge Çetin

Next Generation AAV-F Capsid gene therapy rescues disease pathology in a model of Pyruvate Dehydrogenase Complex Deficiency

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.

Mifepristone alone and in combination with scAAV9-SMN1 gene therapy improves disease phenotypes in Smn2B/- spinal muscular atrophy mice

This study shows that mifepristone, a glucocorticoid antagonist, improves SMA disease phenotypes in mice, both alone and when combined with SMN gene therapy.