Quantitative Systems Pharmacology Model of Sickle Cell Disease and Response to Gene Editing Therapy to Support Clinical Development of SAR445136 (BIVV003)

Sickle cell disease (SCD) is caused by a mutation in the β-globin gene that produces abnormal hemoglobin (HbS), leading to clinical manifestations such as painful vaso-occlusive crises, anemia, and shortened lifespan due to organ damage. SAR445136 (BIVV003) is a zinc finger nuclease ex vivo gene edi...

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Veröffentlicht in:	Blood 2021-11, Vol.138 (Supplement 1), p.1860-1860
Hauptverfasser:	Kaddi, Chanchala, Holz, Danielle, Tao, Mengdi, Galeon, Isobelle, Reiner, David, Rendo, Pablo, Zaph, Susana
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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