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Research in the Davidson Laboratory is focused on inherited genetic diseases that cause central nervous system dysfunction. There are several dynamic research projects underway in the lab. The lab is investigating recessive, childhood-onset neurodegenerative diseases, such as the lysosomal storage diseases mucopolysaccharidoses and Battens disease. The research team is also researching dominant genetic diseases, specifically the CAG repeat disorders, Huntington's disease and spinal cerebellar ataxia. In addition, the lab aims to better understand how changes in the transcriptome impact neural development and neurodegenerative disease processes.
The lab's research on childhood onset neurodegenerative diseases is focused on experiments to better understand the biochemistry and cell biology of proteins deficient in these disorders, and to develop small molecule- or gene therapy-based strategies for therapy. In recent work, the lab demonstrated that the application of recombinant viral vectors to various models of storage disease reversed CNS deficits and improved life span. The research team continues its development of novel vector systems to improve therapeutic outcomes.
Another area of research focus in the lab is the development of therapies for dominant disorders. This field presents an exciting challenge that requires the dominant disease allele be silenced. To approach this, the team has developed reagents for expressing inhibitory RNAs or editing machinery (e.g., CrispR/Cas9 approaches) in vivo to improve disease phenotypes in relevant animal models.
Finally, the team in the Davidson Lab investigates how the transcriptome is altered in neurological diseases. By evaluating splicing changes they have discovered novel players in disease pathogenesis that include noncoding RNAs and RNA binding proteins. This work is revealing new pathways of pathogenesis and novel targets for therapy.
Chief Scientific Strategy Officer
Dr. Davidson works to understand the molecular basis of childhood onset neurodegenerative diseases and the development of gene and small molecule therapies for treatment. She also focuses on how noncoding RNAs participate in neural development and neurodegenerative disease processes, and how they can be harnessed for therapies.