Lentiviral Gene Therapy for Severe Leukocyte Adhesion Deficiency Type 1

Claire Booth¹, Julián Sevilla, Elena Almarza+28

• ¹University College London Great Ormond Street Institute of Child Health (C.B., K.C., K.G., A.J.T.); and Great Ormond Street Hospital NHS Foundation Trust (C.B., G.O., J.X.-B.), London, England; Hematología y Hemoterapia, Fundación para la Investigación Biomédica, Hospital Infantil Universitario Niño Jesús (HIUNJ) (J.S., J.Z.); and Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII) (J.S., E.A., J.A.B.), Madrid; Rocket Pharmaceuticals, Cranbury, NJ (E.A., M.C.-L., G.C., E.N., S.K., G.R.R., K.P., G.S., J.D.S.); Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT) (E.A., J.A.B.); and Unidad Mixta de Terapias Avanzadas, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD)/CIEMAT (E.A., J.A.B.), Madrid; University of California, Los Angeles, Los Angeles (C.Y.K., D.T., A.F., S.D.O., C.B., C.J., T.B.M., D.B.K.); and Department of Clinical Pharmacy, University of California, San Francisco, San Francisco (J.L.-B.), CA; Institute of Experimental Hematology, Hannover Medical School, Hannover, Germany (A.L.B., P.J.-N., A.S., M.R.); Immunology Department, Great Ormond Street Hospital, London, England (K.G.); and Division of Hematology/Oncology, Boston Children's Hospital, Harvard Medical School, Boston, MA (A.S.).