“In this study, we showed that a single ICV injection of AAV9-Syn1-WWOX in Wwox-null neonates rescues the phenotypes associated with WWOX deficiency including growth retardation, hypoglycemia, hypomyelination, seizures and premature lethality.” says Prof. Aqeilan. “Additionally, treated mice were fertile and exhibited normal behaviour” he added.
This proof-of-concept preclinical assessment provides hope for WOREE and SCAR12 syndrome patients. In this context, the Aqeilan lab would like to thank all the patients, their families, and WWOX Foundation for their continuous support and inspiration.”
To the paper
To Prof. Aqeilan
Congratulations to Prof. Rami Aqeilan and his post-doctoral researcher Dr. Sri Repudi for their new paper published in EMBO Molecular Medicine on the preclinical assessment of WWOX gene therapy as a possible treatment for WOREE syndrome.