Dr. Nicolas Wein and his team join TaRGeT lab
He will work on developing therapeutic vector for neuromuscular disorders using RNA and DNA editing tools.
TaRGeT's gene therapy for neuromuscular diseases program is being strengthened with the arrival of a new researcher. Nicolas Wein and his team are joining our laboratory to work specifically on exon skipping and therapeutic delivery routes for various neuromuscular applications.
We are glad to have Nicolas on board after his extensive experience at Nationwide Children's Hospital.
Since 2016, his laboratory in the USA studied the mechanisms of pathogenesis of neuromuscular disorders (e.g. Duchenne Muscular dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1). Their long-term projects focus on RNA and DNA editing and gene transfer as therapies for neuromuscular disorders, but they will also include more fundamental research on muscle function. In particular, his lab uses the newest technologies, such as virus-mediated exon-skipping and the CRISPR/Cas9 genome editing tool. They are also investigating alternative way to inject these therapies.
His team started this year at TaRGeT where they will continue to work on developing therapeutic vector for neuromuscular disorders using RNA and DNA editing tools.