Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1

C Claire Booth (University College London Great Ormond Street Institute of Child Health, London) J Julián Sevilla (Hematología y Hemoterapia, Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid) E Elena Almarza (Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid) C Caroline Y. Kuo (University of California, Los Angeles, Los Angeles) J Josune Zubicaray (Hematología y Hemoterapia, Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid) D Dayna Terrazas (Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles) G Grainne O’Toole (Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London) M Maria Chitty-Lopez (Rocket Pharmaceuticals, Cranbury, NJ) G Grace Choi (Rocket Pharmaceuticals, Cranbury, NJ) E Eileen Nicoletti (Rocket Pharmaceuticals, Cranbury, NJ) J Janel Long-Boyle (Department of Clinical Pharmacy, University of California, San Francisco, San Francisco) A Augustine Fernandes (Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles) K Kritika Chetty (University College London Great Ormond Street Institute of Child Health, London) S Satiro De Oliveira (Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles) C Crystal Banuelos (University of California, Los Angeles, Los Angeles) J Jinhua Xu-Bayford (Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London) A Antonella Lucía Bastone (Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany) P Philipp John-Neek (Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany) C Connie Jackson (University of California, Los Angeles, Los Angeles) T Theodore B. Moore (Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles) K Kimberly Gilmour (Great Ormond Street Hospital for Children NHS Trust, London) A Axel Schambach (Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany) M Michael Rothe (Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany) S Sanchali Kasbekar (Rocket Pharmaceuticals, Cranbury, NJ) G Gayatri R. Rao (Rocket Pharmaceuticals, Cranbury, NJ) K Kinnari Patel (Rocket Pharmaceuticals, Cranbury, NJ) G Gaurav Shah (Rocket Pharmaceuticals, Cranbury, NJ) A Adrian J. Thrasher (University College London Great Ormond Street Institute of Child Health, London) J Juan A. Bueren (Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid) J Jonathan D. Schwartz (Rocket Pharmaceuticals, Cranbury, NJ) D Donald B. Kohn (Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles)

Article Details

Volume / Issue Vol. 392, Issue 17
Published May 01, 2025
Pages 1698-1709
ISSN 0028-4793
Publisher Massachusetts Medical Society

Journal Info

New England Journal of Medicine

Massachusetts Medical Society

ISSN: 0028-4793 Social Sciences

Authors (31)

C

Claire Booth

University College London Great Ormond Street Institute of Child Health, London

J

Julián Sevilla

Hematología y Hemoterapia, Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid

E

Elena Almarza

Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid

C

Caroline Y. Kuo

University of California, Los Angeles, Los Angeles

J

Josune Zubicaray

Hematología y Hemoterapia, Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ), Madrid

D

Dayna Terrazas

Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles

G

Grainne O’Toole

Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London

M

Maria Chitty-Lopez

Rocket Pharmaceuticals, Cranbury, NJ

G

Grace Choi

Rocket Pharmaceuticals, Cranbury, NJ

E

Eileen Nicoletti

Rocket Pharmaceuticals, Cranbury, NJ

J

Janel Long-Boyle

Department of Clinical Pharmacy, University of California, San Francisco, San Francisco

A

Augustine Fernandes

Department of Microbiology, Immunology, and Molecular Genetics, David Geffen School of Medicine, University of California, Los Angeles

K

Kritika Chetty

University College London Great Ormond Street Institute of Child Health, London

S

Satiro De Oliveira

Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles

C

Crystal Banuelos

University of California, Los Angeles, Los Angeles

J

Jinhua Xu-Bayford

Department of Paediatric Immunology and Gene Therapy, Great Ormond Street Hospital for Sick Children NHS Foundation Trust, London

A

Antonella Lucía Bastone

Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany

P

Philipp John-Neek

Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany

C

Connie Jackson

University of California, Los Angeles, Los Angeles

T

Theodore B. Moore

Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles

K

Kimberly Gilmour

Great Ormond Street Hospital for Children NHS Trust, London

A

Axel Schambach

Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany

M

Michael Rothe

Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany

S

Sanchali Kasbekar

Rocket Pharmaceuticals, Cranbury, NJ

G

Gayatri R. Rao

Rocket Pharmaceuticals, Cranbury, NJ

K

Kinnari Patel

Rocket Pharmaceuticals, Cranbury, NJ

G

Gaurav Shah

Rocket Pharmaceuticals, Cranbury, NJ

A

Adrian J. Thrasher

University College London Great Ormond Street Institute of Child Health, London

J

Juan A. Bueren

Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid

J

Jonathan D. Schwartz

Rocket Pharmaceuticals, Cranbury, NJ

D

Donald B. Kohn

Department of Pediatrics, David Geffen School of Medicine, University of California, Los Angeles, Los Angeles