SCIWORTHY.COM (PICTURE) WHICH HAS GREAT IMAGES AND A GREAT SUMMARY WEBSITE OF THIS TOPIC!
Gene Therapy for DYT-1 Dystonia and other diseases?
We sat down with one the experts on gene therapy for DYT-1 dystonia and other central nervous system diseases and we picked his brain on this topic. The interview was fascinating!
Who is Edgar Rodriguez?
Dr. Edgardo (Edgar) Rodríguez-Lebrón has 20 years of experience in the gene therapy space with a focus on diseases of the nervous system. He has engineered and developed gene-based therapies to prevent or reverse neurodegenerative processes caused by the accumulation of toxic mutant proteins. He is currently Scientific Advisor to Tyler's Hope, Chief Science Officer of Andante Biologics and a co-founder of early-stage biotech companies developing molecular therapies for CNS diseases. He holds an Adjunct Faculty appointment at the University of Florida where he trained as a graduate student. Go Gators!
Often, diseases that affect the brain are caused by genetic mutations that prevent the normal function of a gene. In other cases, genetic mutations can cause a gene to ‘gain’ a new function that is toxic to the brain.
Gene therapies are a class of medicines designed to specifically (i) replace a missing genetic function, (ii) block a ‘gained’ toxic genetic function, or (iii) limit or enhance cellular pathways to support overall brain function.
What is a viral-based gene therapy?
What makes AAV a desirable vector for CNS-targeted gene therapies?
What are some recent major developments in AAV CNS gene therapy?
Recent developments include:
What are some of the major hurdles still being faced by AAV-based gene therapies?
How can an AAV-based gene therapy be used in DYT1 dystonia?
Applications to DYT-1:
What is on the horizon for AAV-based gene therapies as it applies to DYT1?
On the horizon: