Neuromuscular Home Page 2022-2024 Form

Instructions and help about Neuromuscular Clinical Laboratory

[Music] so the goal of our lab is to identify therapies for neuromuscular disorders and these are disorders that affect muscles or nerves my lab is specifically focused on muscular dystrophies so looking for genetic treatments specifically Gene therapies muscular dystrophies are genetically defined disorders that affect muscle so they cause muscles to become weak and they eventually become so weak that a person can't walk and eventually in some of these disorders they can't breathe and they'll eventually pass due to this disorder the muscular dystrophies are rare disorders and very few of them have any treatments at all so there's a really unmet need High unmet need for for treatments because we know the genetic cause we can act on that knowledge and and do gene therapy to help these patients for the gene therapies there are two main ways that people approach these diseases one is Gene replacement where you put back a gene that was mutant or missing and the second is Gene editing where