Results for Dystonia

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Dr. Gonzalez-Alegre's long-range research goal is to advance the application of precision medicine in the neurology clinic. His research focus revolves around genetic disorders that affect the brain, spanning from the diagnosis of novel genetic disease in the clinic to the identification of novel molecular targets using disease models and the design of early-phase human clinical trials.

E-mail:

gonzalezap [at] email.chop.edu

View Pedro Gonzalez-Alegre's Profile

Benjamin C. Kennedy, MD

Dr. Kennedy's current research focuses on epilepsy genetics and epilepsy surgery, including stereo-EEG, hemispherotomy, corpus callosotomy, and vagal nerve stimulation.

E-mail:

kennedybc [at] email.chop.edu

View Benjamin C. Kennedy's Profile

Scientists Explore Gene Silencing as Novel Approach to Dystonia

Published on

Apr 24, 2015

in Cornerstone Blog

Scientists at The Children’s Hospital of Philadelphia are comparing two molecular therapy techniques for Dystonia — RNA interference (RNAi) and antisense oligonucleotides (ASOs).

Gonzalez-Alegre Laboratory

The Gonzalez-Alegre Lab studies inherited genetic diseases of the nervous system, such as dystonia, Huntington’s disease, and inherited ataxias. The lab uses cellular and animal models as well as clinical observation to better understand the genetic causes and molecular mechanisms of these diseases, develop potential therapies, and assess the efficacy of these treatments.