A Novel Retinal Gene Therapy Strategy for Batten Disease and Beyond

Affiliations

Maura Schwartz: The Abigail Wexner Research Institute at Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, OH 43205, USA
Alex Campbell: The Abigail Wexner Research Institute at Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, OH 43205, USA
Joseph Caporale: The Abigail Wexner Research Institute at Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, OH 43205, USA
Megan Baird: The Abigail Wexner Research Institute at Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, OH 43205, USA
Isabella Palazzo: The Abigail Wexner Research Institute at Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, OH 43205, USA
Shibi Likhite: The Abigail Wexner Research Institute at Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, OH 43205, USA
Andrew Fischer: The Abigail Wexner Research Institute at Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, OH 43205, USA
Kathrin Meyer: The Abigail Wexner Research Institute at Nationwide Children’s Hospital, Center for Gene Therapy, Columbus, OH 43205, USA

Abstract

Batten Disease is a fatal lysosomal storage disorder characterized by cognitive and [...]