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Matt Porteus, MD PhD
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Journal Articles

First Report of Non-Genotoxic Conditioning with JSP191 (anti-CD117) and Hematopoietic Stem Cell Transplantation in a Newly Diagnosed Patient with Severe Combined Immune Deficiency

Rajni Agarwal, MD, Kenneth I. Weinberg, MD, Hye-Sook Kwon, PhD, Anne Le, Janel R Long-Boyle, PharmD, PhD, Donald B. Kohn, MD, Kathryn Bradford, MD, Satiro De Oliveira, MD, Alice Bertaina, MD PhD, Agnieszka Czechowicz, MDPhD, Matt Porteus, MD PhD, David C Shyr, MD, Katja G Weinacht, MD PhD, Ami J Shah, MD, Melissa Mavers, MD PhD, Elisabeth Merkel, RN, Janice W Brown, MD, Christopher C. Dvorak, MD, Robertson Parkman, MD, Maria Grazia Roncarolo, MD, Judith A Shizuru, MD PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 10.
https://doi.org/10.1182/blood-2020-137762
Published: 2020
Journal Articles

Correction of the Energetic Defects in Pyruvate Kinase Deficiency through Genome Editing in Hematopoietic Stem and Progenitor Cells

Sara Fañanas-Baquero, Oscar Quintana Bustamante, PhD, Daniel P. Dever, PhD, Omaira Alberquilla, Rebeca Sanchez-Dominguez, PhD, Joab Camarena, BS, Rolf Turk, José Luis López Lorenzo, MD, Paola Bianchi, BSc, PhD, Mark Behlke, MD PhD, Juan A Bueren, PhD, Matt Porteus, MD PhD, José C Segovia, PhD
Journal: Blood
Blood (2020) 136 (Supplement 1): 29.
https://doi.org/10.1182/blood-2020-139117
Published: 2020