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Amyotrophic Lateral Sclerosis, C9ORF72-associated

Amyotrophic Lateral Sclerosis, C9ORF72-associated

This image shows the presence of nuclear RNA foci.

What is Amyotrophic lateral sclerosis?

Amyotrophic lateral sclerosis (ALS), also known as Charcot disease after the physician who first described it, and Lou Gehrig’s disease in the US after a famous baseball player who suffered from ALS, is a neurodegenerative disorder that primarily affects the motor neurons.

Clinically this leads to progressive muscle weakness, progressive paralysis and ultimately death within three to five years post-diagnosis. There is currently no cure for ALS. Despite the number of ongoing clinical trials, the only FDA-approved treatment to date is riluzole, which only increases patient survival by about 3 months. There is therefore an unmet medical need for this fatal disease, and several novel therapeutics are being actively investigated. One of the proposed strategies is gene therapy.

Our therapeutic strategy: silencing C9ORF72

Mutations in the C9ORF72 (chromosome 9, open reading frame 72) gene have been associated with ALS. We aim to silence C9ORF72 using an artificial miRNA. In this research area we are partnering with Dr. Robert H. Brown Jr. You can visit his website here.  

  

Let's have the next breakthrough together! 

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