Clinical outcomes and sustained biochemical engraftment following ex-vivo autologous stem cell gene therapy for mucopolysaccharidosis type IIIA
- Resource Type
- Abstract
- Authors
- Jones, Simon; Kinsella, Jane; Holley, Rebecca J.; Potter, Jane; Booth, Claire; Buckland, Karen; Rust, Stewart; Bromley, Rebecca; Church, Heather J.; Lee, Helena; Ford, Laura; Learmonth, Yuko Ishikawa; Searle, Rachel; Ellison, Stuart M.; Wynn, Robert; Bigger, Brian
- Source
- In Molecular Genetics and Metabolism February 2024 141(2)
- Subject
- Language
- ISSN
- 1096-7192