Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease
- Resource Type
- Article
- Authors
- Dogan, Yildirim; Barese, Cecilia N.; Schindler, Jeffrey W.; Yoon, John K.; Unnisa, Zeenath; Guda, Swaroopa; Jacobs, Mary E.; Oborski, Christine; Maiwald, Tim; Clarke, Diana L.; Schambach, Axel; Pfeifer, Richard; Harper, Claudia; Mason, Chris; van Til, Niek P.
- Source
- In Molecular Therapy - Methods & Clinical Development 8 December 2022 27:464-487
- Subject
- Language
- ISSN
- 2329-0501