CRISPR-Cas9 | CHOP Research Institute


Published on
May 10, 2024
This week, honors for innovative CHOP investigators, early results of gene editing for inherited blindness, plus ASGCT and PAS meeting highlights.
Published on
Jun 16, 2021
Meet our featured Faculty Spotlight scientist for June, Will Bailis, PhD.

Our research on childhood onset neurodegenerative diseases is focused on better understanding the biochemistry and cell biology of proteins deficient in these disorders and developing small molecule or gene therapy-based strategies for therapy.

The Human Pluripotent Stem Cell Core provides services for the development of novel, human cell-based experimental models for elucidating the pathophysiology of genetic disorders.

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.

davidsonbl [at]