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    Journal Articles

    Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer Results in Enriched Biallelic Editing, Increased Fetal Hemoglobin, and Reduced Sickling in Erythroid Cells Derived from Sickle Cell Disease Patients

    Samuel Lessard, PhD, Pauline Rimmele, PhD, Hui Ling, PhD, Kevin Moran, MSc, Benjamin Vieira, BS, Yi-Dong Lin, PhD, Vu Hong, PhD, Andreas Reik, PhD, Denny Dang, Naoya Uchida, MD, John F. Tisdale, MD, Pablo Rendo, MD, Ahmed Daak, PhD, Alexandra Hicks, PhD
    Journal: Blood
    Blood (2019) 134 (Supplement_1): 974.
    https://doi.org/10.1182/blood-2019-126048
    Published: 2019
    Journal Articles

    Ex Vivo Gene-Edited Cell Therapy for Sickle Cell Disease: Disruption of the BCL11A Erythroid Enhancer with Zinc Finger Nucleases Increases Fetal Hemoglobin in Plerixafor Mobilized Human CD34+ Cells

    Kevin Moran, MSc, Hui Ling, PhD, Samuel Lessard, PhD, Benjamin Vieira, Vu Hong, PhD, Michael C. Holmes, PhD, Andreas Reik, PhD, Denny Dang, David Gray, Dana Levasseur, PhD, Pauline Rimmele, PhD
    Journal: Blood
    Blood (2018) 132 (Supplement 1): 2190.
    https://doi.org/10.1182/blood-2018-99-116998
    Published: 2018

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