Neuron-specific WWOX gene therapy produces dose-dependent, durable rescue in a model of WWOX-related epileptic encephalopathy
Mustafa Obeid, Rania Akkawi, Srinivasarao Repudi, Prince Kumar Singh, Baraa Abudiab, Takwa Jebara, Allyson Berent, Thomas Brennan, Yael Weiss, Tawfeeq Shekh-Ahmad, Rami I Aqeilan · 2026
Read Neuron-specific WWOX gene therapy produces dose-dependent, durable rescue in a model of WWOX-related epileptic encephalopathy by Mustafa Obeid, Rania Akkawi, Srinivasarao Repudi, Prince Kumar Singh, Baraa Abudiab, Takwa Jebara, Allyson Berent, Thomas Brennan, Yael Weiss, Tawfeeq Shekh-Ahmad, Rami I Aqeilan (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/PMC13343157.txt.
Description
Mustafa Obeid and co-authors design a gene therapy for WWOX-related epileptic encephalopathy in a 2026 mouse study. Children missing working WWOX copies face severe developmental and seizure disorders with no effective treatment, so the team tuned a replacement therapy in mice that lack the gene entirely.
Neuron-only gene expression, driven by the human synapsin promoter in an AAV9 virus, beat broader or glia-targeted designs. Dropping an extra control element allowed careful dosing, and the best dose restored survival, growth, behavior, and fertility close to normal. Treated brains gained myelin and calmed inflammation, and early treatment in the first five days of life gave lasting rescue, setting design rules for patient trials.
Catalog record
| CREATOR | Mustafa Obeid, Rania Akkawi, Srinivasarao Repudi, Prince Kumar Singh, Baraa Abudiab, Takwa Jebara, Allyson Berent, Thomas Brennan, Yael Weiss, Tawfeeq Shekh-Ahmad, Rami I Aqeilan |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 61520 |
| LICENSE | https://creativecommons.org/licenses/by/4.0 |
| RIGHTS | CC BY |
| SOURCE | Europe PMC |