Accept Cookies?
Provided by OpenGlobal E-commerce

Please wait while your page loads ...

Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich's Ataxia

In this review, the authors provide an overview on the current and emerging prospects of gene therapy for FRDA, with specific focus on advantages of CRISPR/Cas9-mediated gene editing of FXN as a viable option to restore endogenous frataxin expression. They also assess the potential of ex vivo gene editing in hematopoietic stem and progenitor cells as a potential autologous transplantation therapeutic option and discuss its advantages in tackling FRDA-specific safety aspects for clinical translation.

Read the Full article here


Scientific News Archives

Tagged in
Scientific News


Privacy Policy      Service Terms      Contact      Charity Navigator