Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1
In this study, lentiviral vector-transduced autologous CD34+ HSCT was successful in treating severe LAD-I. (Funded by Rocket Pharmaceuticals and the California Institute for Regenerative Medicine; ClinicalTrials.gov numbers, NCT03812263 and NCT06282432.).
Authors
- Augustine Fernandes
- Claire Booth (ORCID: https://orcid.org/0000-0002-2626-5037)
- Philipp John-Neek
- Elena Almarza (ORCID: https://orcid.org/0000-0002-4414-3455)
- Juan A. Bueren (ORCID: https://orcid.org/0000-0002-3228-7013)
- Kinnari Patel (ORCID: https://orcid.org/0000-0001-6192-6526)
- Caroline Y. Kuo (ORCID: https://orcid.org/0000-0002-9302-0187)
- Adrian J. Thrasher (ORCID: https://orcid.org/0000-0002-6097-6115)
- Kritika Chetty (ORCID: https://orcid.org/0000-0003-1462-3623)
- Satiro De Oliveira (ORCID: https://orcid.org/0000-0002-8181-7316)
- Maria Chitty-Lopez
- Josune Zubicaray (ORCID: https://orcid.org/0000-0003-4115-9598)
- Donald B. Kohn (ORCID: https://orcid.org/0000-0003-1840-6087)
- Gráinne O’Toole
- Axel Schambach (ORCID: https://orcid.org/0000-0003-2743-0070)
- Theodore B. Moore (ORCID: https://orcid.org/0000-0002-7909-610X)
- Antonella Lucía Bastone (ORCID: https://orcid.org/0000-0003-0356-2909)
- Julián Sevilla (ORCID: https://orcid.org/0000-0002-6852-1860)
- Carmen A. Bañuelos
- Sanchali Kasbekar
- Connie Jackson
- Jonathan Schwartz (ORCID: https://orcid.org/0000-0001-8965-6337)
- Jinhua Xu‐Bayford
- Gayatri R. Rao
- Janel Long‐Boyle
- Kimberly Gilmour (ORCID: https://orcid.org/0000-0003-0285-3958)
- Eileen Nicoletti
- Grace Choi
- Michael Rothe (ORCID: https://orcid.org/0000-0002-6813-4705)
- Gaurav Y. Shah (ORCID: https://orcid.org/0000-0002-0210-9195)
- Dayna Terrazas
Institutions
- Boston Children's Hospital (US)
- Harvard University (US)
- University of California, Los Angeles (US)
- University of California, San Francisco (US)
- Great Ormond Street Hospital (GB)
- Great Ormond Street Hospital for Children NHS Foundation Trust (GB)
- Instituto de Salud Carlos III (ES)
- Medizinische Hochschule Hannover (DE)
- Centre for Biomedical Network Research on Rare Diseases (ES)
- Hospital Infantil Universitario Niño Jesús (ES)
- Hospital Universitario Fundación Jiménez Díaz (ES)
- Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas (ES)
- University College London (GB)
Publication Details
- Journal
- New England Journal of Medicine
- Published
- 2025-05-01
- DOI
- https://doi.org/10.1056/nejmoa2407376
- Citations
- 9
- Primary Topic
- Microfluidic and Bio-sensing Technologies
- Type
- article
- Field-Weighted Citation Impact
- 6.07
Funders
- California Institute for Regenerative Medicine