Katherine A White, Hemanth R Nelvagal, Timothy A Poole, Bin Lu, Tyler B Johnson, Samantha Davis, Melissa A Pratt, Jon Brudvig, Ana B Assis, Shibi Likhite, Kathrin Meyer, Brian K Kaspar, Jonathan D Cooper, Shaomei Wang, Jill M Weimer
Batten disease is a family of rare, fatal, neuropediatric diseases presenting with memory/learning decline, blindness, and loss of motor function. Recently, we reported the use of an AAV9-mediated gene therapy that prevents disease progression in a mouse model of CLN6-Batten disease ( Cln6 nclf ), restoring lifespans in treated animals. Despite the success of our viral-mediated gene therapy, the dosing strategy was optimized for delivery to the brain parenchyma and may limit the therapeutic potential to other disease-relevant tissues, such as the eye...
March 12, 2021: Molecular Therapy. Methods & Clinical Development