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Journal Articles

Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice

Azadeh Kia, PhD, Jenny McIntosh, PhD, Cecilia Rosales, PhD, Paniz Hosseini, Rose Sheridan, Justyna Spiewak, Kevin Mills, Romuald Corbau, PhD, Amit C Nathwani, MD PhD
Journal: Blood
Blood (2018) 132 (Supplement 1): 2209.
https://doi.org/10.1182/blood-2018-99-116632
Published: 2018
Journal Articles

Defective Function of KCa3.1 Channels in Lysosomal Storage Disorders

Pilar Giraldo, PhD, Jorge J Cebolla, fellow, Pilar Irun, PhD, Laura Lopez de Frutos, MSc, Marcio Andrade-Campos, PhD, Ralf Köhler, PhD
Journal: Blood
Blood (2017) 130 (Supplement 1): 3562.
https://doi.org/10.1182/blood.V130.Suppl_1.3562.3562
Published: 2017
Journal Articles

Lysosomal Newborn Screening in a Cohort in Mexican Population-

Juana Ines Navarrete, MD
Journal: Blood
Blood (2015) 126 (23): 5581.
https://doi.org/10.1182/blood.V126.23.5581.5581
Published: 2015
Journal Articles

BMT Doubles The Lifespan Of A Mouse Model Of Farber Disease and Corrects Hematopoietic Defects

Shaalee Dworski, MSc, Alexandra Berger, PhD, Caren Furlonger, Christopher J. Paige, PhD, Jeffrey A. Medin, PhD
Journal: Blood
Blood (2013) 122 (21): 1997.
https://doi.org/10.1182/blood.V122.21.1997.1997
Published: 2013
Journal Articles

Generation and Implementation of a Novel Murine Xenograft Model for Evaluating Human Hematopoietic Cell-Targeted Gene Therapies for Fabry Disease.

Natalia A. Pacienza, PhD, Nobuo Mizue, PhD, Makoto Yoshimitsu, MD, Matthew Scaife, BSc, Ronan Foley, MD, FRCPC, Jeffrey A. Medin, PhD
Journal: Blood
Blood (2009) 114 (22): 3576.
https://doi.org/10.1182/blood.V114.22.3576.3576
Published: 2009
Journal Articles

Use of MGMT Resistance to Enrich for Transduced Hematopoietic Cells in Gene Therapy for Fabry Disease.

Natalia A. Pacienza, PhD, Nobuo Mizue, PhD, Matthew Scaife, BSc, Ronan Foley, MD, FRCPC, Jeffrey A. Medin, PhD
Journal: Blood
Blood (2009) 114 (22): 3584.
https://doi.org/10.1182/blood.V114.22.3584.3584
Published: 2009
Journal Articles

Haplotype Block for Inflammatory Cytokine Gene Polymorphisms in Fabry Disease.

Deborah Elstein, PhD, Rachael Safyan, Catharina Whybra, MD, Michael Beck, MD, Gheona Altarescu, MD
Journal: Blood
Blood (2006) 108 (11): 3834.
https://doi.org/10.1182/blood.V108.11.3834.3834
Published: 2006
Journal Articles

Elevated Counts of Circulating Endothelial Microparticles in Pediatric Fabry Patients Decreased after Enzyme Replacement Therapy.

Monique P. Gelderman-Fuhrmann, PhD, Raphael Schiffmann, MD, Jan Simak, PhD
Journal: Blood
Blood (2006) 108 (11): 1818.
https://doi.org/10.1182/blood.V108.11.1818.1818
Published: 2006
Journal Articles

Functional Engraftment of Retrovirally Transduced and Pre-Selected Hematopoietic Cells into Partially Ablated Recipient Fabry Mice.

Jeffrey A. Medin, PhD, Sheng-Ben Liang, MD, PhD, Armando Poeppl, Makoto Yoshimitsu, MD, Jagdeep S. Walia, MBBS, Jianhui Cai, MD, PhD, Vanessa I. Rasaiah, Daniel H. Fowler, MD
Journal: Blood
Blood (2005) 106 (11): 3023.
https://doi.org/10.1182/blood.V106.11.3023.3023
Published: 2005
Journal Articles

Novel Mechansims for P k (Gb 3 ) Inhibition of HIV Infection.

Nicole Lund, Clifford A. Lingwood, PhD, Jeffrey A. Medin, PhD, Martin L. Olsson, MD, PhD, Cyril Levene, MD, Vered Yahalom, MD, Donald R. Branch, PhD
Journal: Blood
Blood (2005) 106 (11): 1430.
https://doi.org/10.1182/blood.V106.11.1430.1430
Published: 2005
Journal Articles

Autoimmunity and Hypercoagulability in Anderson Fabry Patients from Argentina.

Mario C. Aggio, MD, Pablo A. Martinez, PhD
Journal: Blood
Blood (2005) 106 (11): 4115.
https://doi.org/10.1182/blood.V106.11.4115.4115
Published: 2005
Journal Articles

Long-Term and Sustained Correction of the α-Galactosidase A Deficiency in Fabry Mice and Patient Cells Receiving Lentivirally Transduced Hematopoietic Stem/Progenitor Cells.

Makoto Yoshimitsu, MD, Christopher Siatskas, PhD, Sheng-Ben Liang, MD, PhD, Vanessa I. Rasaiah, Koji Higuchi, MD, Gary J. Murray, PhD, Toshihiro Takenaka, MD, PhD, Chuwa Tei, MD, PhD, Roscoe O. Brady, MD, Jeffrey A. Medin, PhD
Journal: Blood
Blood (2005) 106 (11): 1285.
https://doi.org/10.1182/blood.V106.11.1285.1285
Published: 2005
Journal Articles

Initiation of a Pre-Clinical Gene Therapy Study in Non-Human Primates Using Lentivectors Targeting Hematopoietic Cells for Correction of Fabry Disease.

Jagdeep S. Walia, MBBS, Makoto Yoshimitsu, MD, Josh D. Silvertown, PhD, Armando Poeppl, Vanessa I. Rasaiah, BSc, Robert E. Donahue, DVM, Robert G. Andrews, MD, J. Andrea McCart, MD, Jeffrey A. Medin, PhD
Journal: Blood
Blood (2005) 106 (11): 5542.
https://doi.org/10.1182/blood.V106.11.5542.5542
Published: 2005
Journal Articles

Thrombophylic Profile of Fabry Patients in Argentina.

Mario C. Aggio, MD, Pablo A. Martinez, PhD
Journal: Blood
Blood (2004) 104 (11): 4048.
https://doi.org/10.1182/blood.V104.11.4048.4048
Published: 2004