Accept Cookies?
Provided by OpenGlobal E-commerce

Please wait while your page loads ...

FARAFARA Cure FA

An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models

Friedreich ataxia (FRDA) is a genetic disease due to increased repeats of the GAA trinucleotide in intron 1 of the frataxin gene. This mutation leads to a reduced expression of frataxin. We have produced an adeno-associated virus (AAV)9 coding for human frataxin (AAV9-hFXN). This AAV was delivered by intraperitoneal (IP) injection to young conditionally knockout mice in which the frataxin gene had been knocked-out in some tissues during embryogenesis by breeding them with mice expressing the Cre recombinase gene under the muscle creatine kinase (MCK) or the neuron-specific enolase (NSE) promoter.

Read More: An AAV9 coding for frataxin clearly improved the symptoms and prolonged the life of Friedreich ataxia mouse models


About the Author

Jen Farmer

Jen Farmer

Executive Director

SHARE

FacebookTwitterLinkedinShare on Google+
Event A.jpg

 

Archived in
  Scientific News


 

 

Tagged in
FARA Scientific News