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Andrea D. Karlen
Andrea D. Karlen
Haematopoiesis
Stem cell
Mucopolysaccharidosis type I
Enzyme assay
Transduction (genetics)
4
Papers
1
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0
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Ex vivo lentiviral transduction of hematopoietic stem cells in mucopolysaccharidosis type II (MPS II) mice achieves high levels of systemic iduronate-2-sulfatase (IDS) enzyme activity and normalization of glycosaminoglycans (GAGs)
2021
Molecular Genetics and Metabolism
Miles Smith
Lalitha R. Belur
Olivia Erlanson
Kelly M. Podetz-Pedersen
Andrea D. Karlen
Jessica McKenzie
Jenn Detellis
Khatuna Gagnidze
Geoff Parsons
Nicholas A. Robinson
Shelby LaBarre
Gina Scarglia
Saumil Shah
Hsing Chen Tsai
R. Scott McIvor
Melissa Bonner
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Effect of supraphysiological alpha-L-iduronidase (IDUA) expression on skeletal manifestations in mucopolysaccharidosis type I (MPS I) mice following ex vivo lentiviral vector transduction of hematopoietic stem cells
2021
Molecular Genetics and Metabolism
Troy C. Lund
Lalitha R. Belur
Avery Huber
Hillary Mantone
Miles Smith
Kelly M. Podetz-Pedersen
Andrea D. Karlen
Elizabeth A Braunlin
Nicholas A. Robinson
Hsing Chen Tsai
R. Scott McIvor
Melissa Bonner
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Comparative systemic and neurologic effectiveness of intravenous and intrathecal AAV9 delivered individually or combined in a murine model of mucopolysaccharidosis type I
2021
Molecular Genetics and Metabolism
Lalitha R. Belur
Avery Huber
Hillary Mantone
Andrea D. Karlen
Miles Smith
Li Ou
Chester B. Whitley
Nicholas Buss
R. Scott McIvor
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Systemic high-level IDUA enzyme activity with correction of neurologic deficit in mucopolysaccharidosis type I mice by lentiviral transduction of hematopoietic stem cells
2020
Molecular Genetics and Metabolism
Lalitha R. Belur
Jessica McKenzie
Kelly M. Podetz-Pedersen
Andrea D. Karlen
Patricia Coutinho de Souza
Shelby LaBarre
Miles Smith
Avery Huber
Jennifer Detellis
Khatuna Gagnidze
Geoff Parsons
Hsing Chen Tsai
R. Scott McIvor
Melissa Bonner
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