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: Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I)

Researchers

Presenter

  • Claire Booth

Principal Investigators

  • Gayatri R. Rao

  • Maria Chitty Lopez

  • Elena Almarza

  • Dayna Terrazas

  • Josune Zubicaray

  • Marta González Vicent

  • Kritika Chetty

  • Grainne O'Toole

  • Jinhua Xu-Bayford

  • Eileen Nicoletti

  • Augustine Fernandes

  • Caroline Kuo

  • Satiro De Oliveira

  • Theodore B. Moore

  • Grace Choi

  • Miriam Zeini

  • Cristina Mesa-Núñez

  • Adrian J. Thrasher

  • Juan A. Bueren

  • Jonathan D. Schwartz

  • Donald B. Kohn

Medical Centers

  • University of California, Los Angeles, Los Angeles, CA

  • Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD), Madrid, Spain

  • Fundación para la investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain

  • UCL Great Ormond Street Institute of Child Health, London, United Kingdom

  • Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT) and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain

Locations

  • United States

  • Spain

  • United Kingdom

Companies

  • N/A

Study Components

Therapeutic Area

  • Genetic Disorder

  • Autoimmune (AI)

Disease

  • Leukocyte adhesion deficiency

Biomarkers

  • N/A

Drug/Treatment

  • Filgrastim

  • Plerixafor

  • Busulfan

Outcome

  • N/A

Study Design

  • Efficacy

  • Safety

Phase

  • I/II

Study Id's

  • N/A

Sponsors

  • N/A

Result

  • N/A

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