Lentiviral gene therapy reverts GPIX expression and phenotype in Bernard-Soulier syndrome type C.

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Title: Lentiviral gene therapy reverts GPIX expression and phenotype in Bernard-Soulier syndrome type C.
Authors: Martinez-Navajas G; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain.; University of Granada, Department of Biochemistry and Molecular Biology I, Faculty of Science, Avenida Fuentenueva S/n, 18071 Granada, Spain., Ceron-Hernandez J; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain.; University of Granada, Department of Biochemistry and Molecular Biology I, Faculty of Science, Avenida Fuentenueva S/n, 18071 Granada, Spain., Simon I; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain.; University of Granada, Department of Biochemistry and Molecular Biology I, Faculty of Science, Avenida Fuentenueva S/n, 18071 Granada, Spain., Lupiañez P; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain.; University of Granada, Department of Biochemistry and Molecular Biology I, Faculty of Science, Avenida Fuentenueva S/n, 18071 Granada, Spain., Diaz-McLynn S; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain., Perales S; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain.; University of Granada, Department of Biochemistry and Molecular Biology I, Faculty of Science, Avenida Fuentenueva S/n, 18071 Granada, Spain.; Instituto de Investigación Biosanitaria Ibs.GRANADA, Granada, Spain., Modlich U; Department of Gene and Cell Therapy, Institute of Regenerative Medicine, University of Zürich, Wagistrasse 12, 8952 Schlieren-Zürich, Switzerland., Guerrero JA; Department of Haematology, University of Cambridge, Cambridge, UK.; National Health Service Blood and Transplant, Cambridge Biomedical Campus, Cambridge, UK., Martin F; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain.; University of Granada, Department of Biochemistry and Molecular Biology III and Immunology, Faculty of Medicine, Avenida Ilustracion S/n, 18016 Granada, Spain., Sevivas T; Serviço de Sangue, Medicina Transfusional e Imunohemoterapia Do Centro Hospitalar e Universitário de Coimbra, Coimbra, Portugal., Lozano ML; Servicio de Hematología y Oncología Médica, Hospital Universitario Morales Meseguer, Centro Regional de Hemodonación, Universidad de Murcia, IMIB-Pascual Parrilla, CIBERER-ISCIII, U765 Murcia, Spain., Rivera J; Servicio de Hematología y Oncología Médica, Hospital Universitario Morales Meseguer, Centro Regional de Hemodonación, Universidad de Murcia, IMIB-Pascual Parrilla, CIBERER-ISCIII, U765 Murcia, Spain.; Grupo Español de Alteraciones Plaquetarias Congénitas (GEAPC), Madrid, Spain., Ramos-Mejia V; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain., Tersteeg C; Laboratory for Thrombosis Research, IRF Life Sciences, KU Leuven Campus Kulak Kortrijk, Kortrijk, Belgium., Real PJ; GENyO, Pfizer-Universidad de Granada-Junta de Andalucia Centre for Genomics and Oncological Research, PTS, Granada, Avenida de la Ilustracion 114, 18016 Granada, Spain.; University of Granada, Department of Biochemistry and Molecular Biology I, Faculty of Science, Avenida Fuentenueva S/n, 18071 Granada, Spain.; Instituto de Investigación Biosanitaria Ibs.GRANADA, Granada, Spain.
Source: Molecular therapy. Nucleic acids [Mol Ther Nucleic Acids] 2023 Jun 12; Vol. 33, pp. 75-92. Date of Electronic Publication: 2023 Jun 12 (Print Publication: 2023).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101581621 Publication Model: eCollection Cited Medium: Print ISSN: 2162-2531 (Print) Linking ISSN: 21622531 NLM ISO Abbreviation: Mol Ther Nucleic Acids Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2162-2531
DOI:10.1016/j.omtn.2023.06.008