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Michael L. Kaufman
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Journal Articles

Correction of the sickle cell disease mutation in human hematopoietic stem/progenitor cells

Megan D. Hoban, Gregory J. Cost, Matthew C. Mendel, Zulema Romero, Michael L. Kaufman, Alok V. Joglekar, Michelle Ho, Dianne Lumaquin, David Gray, Georgia R. Lill, Aaron R. Cooper, Fabrizia Urbinati, Shantha Senadheera, Allen Zhu, Pei-Qi Liu, David E. Paschon, Lei Zhang, Edward J. Rebar, Andrew Wilber, Xiaoyan Wang, Philip D. Gregory, Michael C. Holmes, Andreas Reik, Roger P. Hollis, Donald B. Kohn
Journal: Blood
Blood (2015) 125 (17): 2597–2604.
https://doi.org/10.1182/blood-2014-12-615948
Published: 2015
Includes: Supplemental data
Journal Articles

A Pre-Clinical Model Of Hematopoietic Stem Cell Based Immunotherapy For Cancer Utilizing The NY-ESO-1 T-Cell Receptor and sr39TK PET Reporter / Suicide Gene

Eric H. Gschweng, BS, Melissa N. McCracken, BS, Thinle Chodon, MD, PhD, Richard C Koya, MD, PhD, Michael L Kaufman, MS, Michelle Ho, BS, Navdeep Saini, Roger P. Hollis, PhD, Antoni Ribas, MD, PhD, Satiro N. De Oliveira, MD, Owen N. Witte, MD, Donald B. Kohn, MD
Journal: Blood
Blood (2013) 122 (21): 2020.
https://doi.org/10.1182/blood.V122.21.2020.2020
Published: 2013
Journal Articles

Zinc Finger Nucleases Targeting The β-Globin Locus Drive Efficient Correction Of The Sickle Mutation In CD34 + Cells

Megan D Hoban, BS, Alok V Joglekar, PhD, David Gray, BS, Michael L Kaufman, MS, Fabrizia Urbinati, PhD, Shantha Senadheera, MS, Gregory Cost, PhD, Andreas Reik, PhD, Michael C. Holmes, PhD, Philip D Gregory, PhD, Roger P. Hollis, PhD, Donald B. Kohn, MD
Journal: Blood
Blood (2013) 122 (21): 2904.
https://doi.org/10.1182/blood.V122.21.2904.2904
Published: 2013
Journal Articles

Gene therapy/bone marrow transplantation in ADA-deficient mice: roles of enzyme-replacement therapy and cytoreduction

Denise A. Carbonaro, Xiangyang Jin, Xingchao Wang, Xiao-Jin Yu, Nora Rozengurt, Michael L. Kaufman, Xiaoyan Wang, David Gjertson, Yang Zhou, Michael R. Blackburn, Donald B. Kohn
Journal: Blood
Blood (2012) 120 (18): 3677–3687.
https://doi.org/10.1182/blood-2012-02-408591
Published: 2012
Includes: Supplemental data