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Journal Articles

Repair of the Blood Brain Barrier and Neutrophil Recovery Following HSCT in Cerebral Adrenoleukodystrophy

Troy C Lund, MDMS, PhD, Weston P. Miller, MD, Dave Nascene, MD, Paul Orchard, MD, Ashish O. Gupta, MBBS, MPH
Journal: Blood
Blood (2018) 132 (Supplement 1): 4628.
https://doi.org/10.1182/blood-2018-99-109801
Published: 2018
Journal Articles

Hematopoietic Stem Cell Transplantation for Children with Neurological Diseases Improves the Outcomes and Not Leads to Significant Late Effects — Report from Russian Group

Kirill Kirgizov, MD PhD, Anastasia Borovkova, MD, Elena Skorobogatova, MD PhD, Tatjana Bykova, MD PhD, Alexey A Maschan, MD PhD Professor, Ludmila S Zubarovskaya, MD PhD Professor, Axel R. Zander, MD PhD Professor, Boris V Afanasyev, MD PhD Professor, Alexander Rumyantsev, MD PhD Professor
Journal: Blood
Blood (2017) 130 (Supplement 1): 4526.
https://doi.org/10.1182/blood.V130.Suppl_1.4526.4526
Published: 2017
Journal Articles

Generation of SIN-Lentiviral Vectors for the Treatment of Canavan Disease Using Ex Vivo Transduction of Hematopoietic Target Cells

Michael R. Greene, MD, Vladimir A. Markov, MD, Jeremy S. Francis, PhD, Paola Leone, PhD
Journal: Blood
Blood (2014) 124 (21): 4806.
https://doi.org/10.1182/blood.V124.21.4806.4806
Published: 2014
Journal Articles

Outcomes Of Allogeneic Cord Blood Transplantation For Leukodystrophies; A Joint Study of Eurocord and “Inborn Errors WP-EBMT”

Hol Janna, Annalisa Ruggeri, MD, Vanderson Rocha, MD, Gerard Michel, Mouhab Ayas, MD, Tracey O'Brien, Gergely Krivan, Bordon Victoria, Rik Schots, MD, PhD, Rovelli Attilio, Elorza Izaskun, Jose M Moraleda, Peter J. Shaw, MBBS, MA, Marleen Renard, Karl-Walter Sykora, MD, Reuven Or, MD, Tsila Zuckerman, MD, Manuel Abecasis, Bredius Robbert, M. Akif Yesilipek, Miguel Angel Diaz, MD, PhD, Gennery Andy, Eliane Gluckman, MD, Jaap Jan Boelens, MD PhD
Journal: Blood
Blood (2013) 122 (21): 2101.
https://doi.org/10.1182/blood.V122.21.2101.2101
Published: 2013
Journal Articles

Clinical Study of Allogenetic Hematopoietic Stem Cell Transplantation for Three Cases of Leukodystrophy

Xiaoqin Feng, Chunfu Li, Na Li, Xuedong Wu
Journal: Blood
Blood (2012) 120 (21): 4559.
https://doi.org/10.1182/blood.V120.21.4559.4559
Published: 2012
Journal Articles

Rescue of Lethal Hypophosphatasia Mice by Neonatal Ex Vivo Gene Therapy Using Lentivirally Transduced Bone Marrow Cells.

Osamu Iijima, PhD, Koichi Miyake, MD, PhD, Hanako Sugano-Tajima, MD, PhD, Tsutomu Igarashi, MD, PhD, Chizu Kanokoda, Atsushi Watanabe, MD, PhD, Takashi Shimada, MD, PhD
Journal: Blood
Blood (2012) 120 (21): 3154.
https://doi.org/10.1182/blood.V120.21.3154.3154
Published: 2012
Journal Articles

The 3'-5' Exonuclease, TREX1, Interacts with Poly(ADP-ribose) Polymerase-1 (PARP1) in Response to DNA Damage

Takuya Miyazaki, MD, PhD, Yong-Soo Kim, PhD, Jeong-Heon Yoon, PhD, Hongsheng Wang, MD, PhD, Herbert C. Morse, III, MD
Journal: Blood
Blood (2012) 120 (21): 1046.
https://doi.org/10.1182/blood.V120.21.1046.1046
Published: 2012