First-in-human high dose AAV9 intrathecal gene therapy for paediatric CLN7 disease: a phase 1, open-label, single ascending dose, non-randomised clinical trial
This study was funded by The Batten's Hope Foundation, Mila's Miracle Foundation, Children's Health Dallas and Philanthropic Gifts to UT Southwestern. In addition, Emily R. Nettesheim received funding from NIH training grant 5T32GM131945-03 and Hamza Dahshi was supported in part by NIH award T32 GM152319.
Authors
- Benjamin Greenberg (ORCID: https://orcid.org/0000-0002-2091-8201)
- Steven J. Gray (ORCID: https://orcid.org/0000-0002-6240-8621)
- Hoang H. Nguyen (ORCID: https://orcid.org/0000-0001-6891-9951)
- Hamza Dahshi (ORCID: https://orcid.org/0009-0000-3097-5421)
- Saima Kayani (ORCID: https://orcid.org/0000-0003-1081-6428)
- Emily R. Nettesheim (ORCID: https://orcid.org/0000-0001-8581-1205)
- Souad Messahel (ORCID: https://orcid.org/0009-0006-1491-8455)
- Susan T. Iannaccone (ORCID: https://orcid.org/0000-0003-0851-3516)
- Veronica Bordes Edgar (ORCID: https://orcid.org/0000-0003-1602-0252)
- Samuel Hughes
- Andrea Lowden (ORCID: https://orcid.org/0000-0002-3204-0790)
- Alagar R Muthukumar
- Srinivasan Kalayarasan
- Berge A. Minassian
- G. S. Varadarajan
Institutions
- St. Jude Children's Research Hospital (US)
- Southwestern Medical Center (US)
- Poseida Therapeutics (United States) (US)
- Case Western Reserve University (US)
- The University of Texas Southwestern Medical Center (US)
Publication Details
- Journal
- EBioMedicine
- Published
- 2025-11-27
- DOI
- https://doi.org/10.1016/j.ebiom.2025.106044
- Primary Topic
- Virus-based gene therapy research
- Type
- article
- Field-Weighted Citation Impact
- 0.00