Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Olivier Nègre; Christian Brendel; Geoffrey Parsons; Min Luo; Myriam Armant; Swaroopa Guda; Lauryn Christiansen; Gretchen Lewis; Gábor Veres; Melissa Bonner

doi:10.1182/blood.v130.suppl_1.3557.3557

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Identifier:nobleid.org/w1/20260515/1193BA0F

Type:Journal Article

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Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown, β-Globin Induction and Simultaneous Repression of β-Globin for the Potential Treatment of Sickle Cell Disease

Identifier:nobleid.org/w1/20260515/1193BA0F

Type:Journal Article

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