Gene Silencing | CHOP Research Institute
 

Gene Silencing

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.

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.

E-mail:
davidsonbl [at] chop.edu
Published on
Apr 24, 2015
Scientists at The Children’s Hospital of Philadelphia are comparing two molecular therapy techniques for Dystonia — RNA interference (RNAi) and antisense oligonucleotides (ASOs).