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.).

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Institutions

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

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article

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1

Augustine Fernandes, Claire Booth, Philipp John-Neek, Elena Almarza et al.
9 citations
New England Journal of Medicine
Microfluidic and Bio-sensing Technologies
6.07
article

Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1

Augustine Fernandes, Claire Booth, Philipp John-Neek, Elena Almarza, Juan A. Bueren, Kinnari Patel, Caroline Y. Kuo, Adrian J. Thrasher, Kritika Chetty, Satiro De Oliveira, Maria Chitty-Lopez, Josune Zubicaray, Donald B. Kohn, Gráinne O’Toole, Axel Schambach, Theodore B. Moore, Antonella Lucía Bastone, Julián Sevilla, Carmen A. Bañuelos, Sanchali Kasbekar, Connie Jackson, Jonathan Schwartz, Jinhua Xu‐Bayford, Gayatri R. Rao, Janel Long‐Boyle, Kimberly Gilmour, Eileen Nicoletti, Grace Choi, Michael Rothe, Gaurav Y. Shah, Dayna Terrazas
article en
9 citations

Abstract

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.).

New England Journal of MedicineVol. 392(17)
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)
California Institute for Regenerative Medicine
Openalex Percentile: Top 4%
Microfluidic and Bio-sensing Technologies
6.07
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