Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy.
- DOI
- 10.1016/j.ymthe.2026.09.032
- Published
- 2026 Sep 25
- Container
- Molecular therapy : the journal of the American Society of Gene Therapy
- Publisher
- Not recorded
- Open access
- unknown
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Cite this work
BibTeX
@article{allodium:10.1016/j.ymthe.2026.09.032,
title = {Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy.},
author = {Kinsella JL and Jones SA and Buckland KF and Holley RJ and Lee H and Ogden W and Potter J and Church HJ and Brammeier KL and Tylee KL and Ellison S and Searle R and Booth C and Farzeneh F and Sherwin L and Thrasher AJ and Rust S and Bigger BW and Wynn RF},
year = {2026},
journal = {Molecular therapy : the journal of the American Society of Gene Therapy},
doi = {10.1016/j.ymthe.2026.09.032},
url = {https://doi.org/10.1016/j.ymthe.2026.09.032}
}RIS
TY - JOUR TI - Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy. AU - Kinsella JL AU - Jones SA AU - Buckland KF AU - Holley RJ AU - Lee H AU - Ogden W AU - Potter J AU - Church HJ AU - Brammeier KL AU - Tylee KL AU - Ellison S AU - Searle R AU - Booth C AU - Farzeneh F AU - Sherwin L AU - Thrasher AJ AU - Rust S AU - Bigger BW AU - Wynn RF PY - 2026 JO - Molecular therapy : the journal of the American Society of Gene Therapy DO - 10.1016/j.ymthe.2026.09.032 UR - https://doi.org/10.1016/j.ymthe.2026.09.032 ER -
APA
JL, K., SA, J., KF, B., RJ, H., H, L., W, O., J, P., HJ, C., KL, B., KL, T., S, E., R, S., C, B., F, F., L, S., AJ, T., S, R., BW, B., & RF, W. (2026). Clinical and biochemical correction of a patient with neurodegenerative Mucopolysaccharidosis IIIA using hematopoietic stem cell gene therapy.. Molecular therapy : the journal of the American Society of Gene Therapy. https://doi.org/10.1016/j.ymthe.2026.09.032
Source records
- pubmed · retrieved 2026-09-26T13:25:26.886Z