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Journal Articles

Gene Therapy for Sickle Cell Anemia Using a Modified Gamma Globin Lentivirus Vector and Reduced Intensity Conditioning Transplant Shows Promising Correction of the Disease Phenotype

Punam Malik, MD, Michael Grimley, MD, Charles T. Quinn, MD, Amy Shova, RN, Little Courtney, RN, Carolyn Lutzko, PhD, Theodosia A. Kalfa, MD PhD, Omar Niss, MD, Parinda A Mehta, MD, Sharat Chandra, MBBS,MD, Elke Grassman, Johannes C.M. Van der Loo, PhD, Scott Witting, MS, Diana Nordling, Archana Shreshta, PhD, Sydney Felker, BS, Catherine Terrell, BS, Lilith Reeves, MS, MT(ASCP), Devin Pillis, MS, Loberg Anastacia, Frederic D Bushman, PhD, Jennifer Knight-Madden, Karen Kalinyak, MD, Stella M. Davies, PhD MBBS, MRCP, Monika Asnani, PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 1021.
https://doi.org/10.1182/blood-2018-99-119591
Published: 2018
Journal Articles

Overcoming reprogramming resistance of Fanconi anemia cells

Lars U. W. Müller, Michael D. Milsom, Chad E. Harris, Rutesh Vyas, Kristina M. Brumme, Kalindi Parmar, Lisa A. Moreau, Axel Schambach, In-Hyun Park, Wendy B. London, Kelly Strait, Thorsten Schlaeger, Alexander L. DeVine, Elke Grassman, Alan D'Andrea, George Q. Daley, David A. Williams
Journal: Blood
Blood (2012) 119 (23): 5449–5457.
https://doi.org/10.1182/blood-2012-02-408674
Published: 2012
Includes: Supplemental data
Journal Articles

FAAP20: a novel ubiquitin-binding FA nuclear core-complex protein required for functional integrity of the FA-BRCA DNA repair pathway

Abdullah Mahmood Ali, Arun Pradhan, Thiyam Ramsingh Singh, Changhu Du, Jie Li, Kebola Wahengbam, Elke Grassman, Arleen D. Auerbach, Qishen Pang, Amom Ruhikanta Meetei
Journal: Blood
Blood (2012) 119 (14): 3285–3294.
https://doi.org/10.1182/blood-2011-10-385963
Published: 2012
Includes: Supplemental data
Journal Articles

Gene-Correction Rescues Reprogramming of Fanconi Anemia Fibroblasts and Enables Hematopoietic Differentiation of FA Induced Pluripotent Stem Cells in Vitro and In Vivo

Lars Mueller, MD, Michael D. Milsom, PhD, Chad Harris, Rutesh Vyas, Kristina Brumme, Kalindi Parmar, PhD, Axel Schambach, MD PhD, Elke Grassman, Ph.D., In-Hyun Park, PhD, London Wendy, PhD, Kelly Strait, Thorsten Schlaeger, Ph.D, Alexander L. Devine, Alan D. D'Andrea, MD, George Q. Daley, MD, PhD, David A. Williams, MD
Journal: Blood
Blood (2011) 118 (21): 672.
https://doi.org/10.1182/blood.V118.21.672.672
Published: 2011
Journal Articles

Use of the in Vitro Immortalization Assay to Quantify the Impact of Integration Spectrum and Vector Design on Insertional Mutagenesis

Ute Modlich, DVM, PhD, Julia Sürth, Daniela Zychlinski, PhD, Johann Meyer, PhD, Christian Brendel, Manuel Grez, PhD, Paritha Arumugam, PhD, Punam Malik, MD, Elke Grassman, PhD, Axel Schambach, MD PhD, Christopher Baum, MD
Journal: Blood
Blood (2011) 118 (21): 3123.
https://doi.org/10.1182/blood.V118.21.3123.3123
Published: 2011
Journal Articles

Somatic Gene Therapy for X-Linked Severe Combined Immunodeficiency Using a Self-Inactivating Modified Gammaretroviral Vector Results in An Improved Preclinical Safety Profile and Early Clinical Efficacy in a Human Patient

Sung-Yun Pai, MD, Luigi Daniele Notarangelo, MD, Chad Harris, Federica Cattaneo, MD, Matthew Wladkowski, Myriam Armant, PhD, Grace S. Kao, MD, Matthew Wielgosz, Arthur W. Nienhuis, MD, Ute Modlich, DVM, PhD, Martijn Brugman, PhD, Axel Schambach, MD PhD, Michael Rothe, Susannah I Thornhill, PhD, H. Bobby Gaspar, Kathryn Parsley, PhD, Samantha Cooray, PhD, Elke Grassman, Ph.D., Frederic Bushman, PhD, Sally Shupien, Matias Oleastro, MD, Salima Hacein-Bey-Abina, Alain Fischer, MD, Marina Cavazzana-Calvo, MD, PhD, Punam Malik, MD, Alexandra Filipovich, MD, Donald B. Kohn, MD, Christopher Baum, MD, David A. Williams, MD, Adrian J Thrasher, MD, PhD
Journal: Blood
Blood (2011) 118 (21): 164.
https://doi.org/10.1182/blood.V118.21.164.164
Published: 2011
Journal Articles

Impaired FANCD2 monoubiquitination and hypersensitivity to camptothecin uniquely characterize Fanconi anemia complementation group M

Thiyam Ramsing Singh, Sietske T. Bakker, Sheba Agarwal, Michael Jansen, Elke Grassman, Barbara C. Godthelp, Abdullah Mahmood Ali, Chang-hu Du, Martin A. Rooimans, Qiang Fan, Kebola Wahengbam, Jurgen Steltenpool, Paul R. Andreassen, David A. Williams, Hans Joenje, Johan P. de Winter, Amom Ruhikanta Meetei
Journal: Blood
Blood (2009) 114 (1): 174–180.
https://doi.org/10.1182/blood-2009-02-207811
Published: 2009
Includes: Supplemental data
Journal Articles

Pulmozyme® Treatment of Vectors Produced by Transient Transfection Reduces Residual Plasmid DNA on Human CD34 + Hematopoietic Progenitor Cells without Loss of Transduction or Engraftment Efficiency

Elke Grassman, Ph.D., Tomoyasu Higashimoto, Ph.D., Justin Rohrbach, Danielle Hall, David A Williams, Lilith Reeves, Punam Malik
Journal: Blood
Blood (2008) 112 (11): 4626.
https://doi.org/10.1182/blood.V112.11.4626.4626
Published: 2008