Molecular therapy : the journal of the American Society of Gene Therapy

Evolution of the clinical-stage hyperactive TcBuster transposase as a platform for robust non-viral production of adoptive cellular therapies.

Pub Date : 2024-04-01 DOI: 10.1016/j.ymthe.2024.04.024 Joseph G. Skeate, E. Pomeroy, Nicholas J. Slipek, Bryan J Jones, B. Wick, J. Chang, Walker S. Lahr, Erin M. Stelljes, Xiaobai Patrinostro, Blake Barnes, Trevor Zarecki, Joshua B. Krueger, Jacob E Bridge, Gabrielle M. Robbins, Madeline D McCormick, John R Leerar, Kari T Wenzel, Kathlyn Hornberger, Kirsti Walker, Dalton Smedley, D. Largaespada, Neil Otto, Beau R. Webber, B. Moriarity

The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.

Pub Date : 2022-11-02 DOI: 10.1016/j.ymthe.2022.10.001 Antonio Monaco, Veronica Maffia, Nicolina Cristina Sorrentino, Irene Sambri, Yulia Ezhova, Teresa Giuliano, Vincenzo Cacace, Edoardo Nusco, Maria De Risi, Elvira De Leonibus, Thomas Schrader, Frank-Gerrit Klärner, Gal Bitan, Alessandro Fraldi

Corneal fibrosis abrogation by a localized AAV-mediated inhibitor of differentiation 3 (Id3) gene therapy in rabbit eyes in vivo.

Pub Date : 2022-10-05 DOI: 10.1016/j.ymthe.2022.08.015 Suneel Gupta, Michael K Fink, Duraisamy Kempuraj, Nishant R Sinha, Lynn M Martin, Landon M Keele, Prashant R Sinha, Elizabeth A Giuliano, Nathan P Hesemann, Sudhanshu P Raikwar, Shyam S Chaurasia, Rajiv R Mohan