A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

▪ Background: Leukocyte Adhesion Deficiency-I (LAD-I) is a rare disorder of neutrophil adhesion resulting from ITGB2 mutations encoding for the β2-integrin component, CD18. Severe LAD-I (CD18

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Veröffentlicht in:Blood 2021-11, Vol.138 (Supplement 1), p.2932-2932
Hauptverfasser: Kohn, Donald B., Booth, Claire, Sevilla, Julián, Rao, Gayatri R, Almarza, Elena, Terrazas, Dayna, Nicoletti, Eileen, Fernandes, Augustine, Kuo, Caroline, de Oliveira, Satiro, Moore, Theodore B., Law, Kenneth M, Beard, Brian C, Choi, Grace, Zeini, Miriam, Mesa-Núñez, Cristina, Thrasher, Adrian J., Bueren, Juan A, Schwartz, Jonathan D
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Sprache:eng
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Zusammenfassung:▪ Background: Leukocyte Adhesion Deficiency-I (LAD-I) is a rare disorder of neutrophil adhesion resulting from ITGB2 mutations encoding for the β2-integrin component, CD18. Severe LAD-I (CD18
ISSN:0006-4971
1528-0020
DOI:10.1182/blood-2021-151898