Hematopoietic Stem-Cell Gene Therapy for Cystinosis
In this small study, CTNS-RD-04, an ex vivo gene therapy for cystinosis, had adverse effects that were largely consistent with the myeloablative regimen and underlying disease profile. White-cell cystine levels decreased after therapy. (Funded by the California Institute for Regenerative Medicine and others; ClinicalTrials.gov number, NCT03897361.).
Authors
- Bruce A. Barshop (ORCID: https://orcid.org/0000-0001-9264-3270)
- Susan A. Phillips (ORCID: https://orcid.org/0009-0008-0914-1607)
- Frederic D. Bushman (ORCID: https://orcid.org/0000-0003-4740-4056)
- Ranjan Dohil
- Anne Sawyers
- Julian Midgley
- Jon A. Gangoiti
- Natalie A. Afshari (ORCID: https://orcid.org/0000-0002-6025-5131)
- Nadine Benador (ORCID: https://orcid.org/0000-0001-5204-103X)
- Doris A. Trauner (ORCID: https://orcid.org/0000-0002-1786-7597)
- Sohini Roy
- Edward D. Ball
- Imama Ahmed
- John K Everett
- Beatriz Campo Fernandes
- Hong Liang
- Stephanie Cherqui
- Maulik Panchal
- Donald Kohn
- Katayoon Shayan
Institutions
- Inserm (FR)
- University of California, Los Angeles (US)
- University of Calgary (CA)
- Centre hospitalier national d'ophtalmologie des Quinze-Vingts (FR)
- University of California San Diego (US)
- Rady Children's Hospital-San Diego (US)
- University of Pennsylvania (US)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2026-02-18
- DOI
- https://doi.org/10.1056/nejmoa2506431
- Citations
- 1
- Primary Topic
- Biomedical Research and Pathophysiology
- Type
- article
- Field-Weighted Citation Impact
- 36.00