Screening of Chimeric GAA Variants in a Preclinical Study of Pompe Disease Results in Candidate Vector for Hematopoietic Stem Cell Gene Therapy
Y. Dogan, C. N. Barese, J. W. Schindler, J. K. Yoon, Z. Unnisa, S. Guda, M. E. Jacobs, C. Oborski, D. L. Clarke, A. Schambach, R. Pfeifer, C. Harper, C. Mason, N. P. van Til
This study screens chimeric GAA variants in a preclinical Pompe disease model, identifying a candidate vector for hematopoietic stem cell gene therapy that reduces glycogen accumulation and CNS vacuolation.
bioRxiv · Dec 28, 2021Bioengineering