Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa).

Weinstein DA, Derks TG, Rodriguez-Buritica DF, Ahmad A, Couce ML, Mitchell JJ, Riba-Wolman R, Mount M, Sallago JB, Ross KM, van der Klauw MM, de Boer F, van der Schaaf C, Saavedra H, Martínez-Olmos M, Atanga E, Hosseini A, Mitragotri D, Crombez E

Open source

DOI: 10.1002/jimd.70014
Published: 2025 Mar
Container: Journal of inherited metabolic disease
Publisher: Not recorded
Open access: yes

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Cite this work

BibTeX

@article{allodium:10.1002/jimd.70014,
  title = {Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa).},
  author = {Weinstein DA and Derks TG and Rodriguez-Buritica DF and Ahmad A and Couce ML and Mitchell JJ and Riba-Wolman R and Mount M and Sallago JB and Ross KM and van der Klauw MM and de Boer F and van der Schaaf C and Saavedra H and Martínez-Olmos M and Atanga E and Hosseini A and Mitragotri D and Crombez E},
  year = {2025},
  journal = {Journal of inherited metabolic disease},
  doi = {10.1002/jimd.70014},
  url = {https://doi.org/10.1002/jimd.70014}
}

RIS

TY  - JOUR
TI  - Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa).
AU  - Weinstein DA
AU  - Derks TG
AU  - Rodriguez-Buritica DF
AU  - Ahmad A
AU  - Couce ML
AU  - Mitchell JJ
AU  - Riba-Wolman R
AU  - Mount M
AU  - Sallago JB
AU  - Ross KM
AU  - van der Klauw MM
AU  - de Boer F
AU  - van der Schaaf C
AU  - Saavedra H
AU  - Martínez-Olmos M
AU  - Atanga E
AU  - Hosseini A
AU  - Mitragotri D
AU  - Crombez E
PY  - 2025
JO  - Journal of inherited metabolic disease
DO  - 10.1002/jimd.70014
UR  - https://doi.org/10.1002/jimd.70014
ER  -

APA

DA, W., TG, D., DF, R., A, A., ML, C., JJ, M., R, R., M, M., JB, S., KM, R., MM, V. D. K., F, D. B., C, V. D. S., H, S., M, M., E, A., A, H., D, M., & E, C. (2025). Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa).. Journal of inherited metabolic disease. https://doi.org/10.1002/jimd.70014

Source records

pubmed · retrieved 2026-09-27T15:49:50.463Z