Research in the Davidson Laboratory is focused on inherited genetic diseases that cause central nervous system dysfunction, with a focus on (1) recessive, childhood onset neurodegenerative disease, such as the lysosomal storage diseases mucopolysaccharidoses and Battens disease; (2) dominant genetic diseases, specifically the CAG repeat disorders, Huntington’s disease and spinal cerebellar ataxia; and (3), understanding how changes in the transcriptome impact neural development and neurodegenerative disease processes.
Published on January 17, 2018
Overcoming limitations inherent in sulfamidase to improve mucopolysaccharidosis IIIA gene therapy
Chen Y, Zheng S, Tecedor L, Davidson BL
Molecular Therapy 2018 Jan 17 doi: 10.1016/j.ymthe.2018.01.010 (Abstract)
Chen et al show that not all lysosomal enzymes are created equal. The authors take a lysosomal enzyme with poor secretory properties, and modify it for improved secretion from gene-corrected cells and better uptake into untransduced cells. After AAV-mediated delivery to brain, cross-correction is more efficient, correcting neuropathology and cognition.