CRISPR/Cas technology application on Rare Diseases

CRISPR/Cas technology application on Rare Diseases

2019-04-16
CRISPR/Cas technology application on Rare Diseases

Two open-access reviews have been recently published on the application of this technology for rare diseases therapy studies.

During last years gene therapy has been embraced as the potential tool for the repair of primary mutations underlying diseases, to date, without cure. 

Gene therapy by genome editing requires the certainty on the causative mutation. Accordingly, several studies for development and refinement of tools based on clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein (Cas) prokaryotic RNA-guided nucleases has prompted a large number of therapy-development studies for Rare Diseases.

In this context, the following two open-access reviews on CRISPR/Cas technology application on Rare Diseases have been recenly published in Molecular Diagnosis & Therapy with the participation of ERN-EuroBloodNet experts from Cyprus Institute of Neurology and Genetics

 

Euro Blood Net