Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment

Preclinical studies of efficacy thresholds and tolerability of a clinically ready lentiviral vector for pyruvate kinase deficiency treatment

Pyruvate kinase deficiency (PKD) is a rare autosomal recessive disorder caused by mutations in the PKLR gene. PKD is characterized by non-spherocytic hemolytic anemia of variable severity and may be fatal in some cases during early childhood. Although not considered the standard of care, allogeneic...

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Main Authors: Susana Navarro, Oscar Quintana-Bustamante, Rebeca Sanchez-Dominguez, Sergio Lopez-Manzaneda, Isabel Ojeda-Perez, Aida Garcia-Torralba, Omaira Alberquilla, Kenneth Law, Brian C. Beard, Antonella Bastone, Michael Rothe, Mariela Villanueva, Juan C. Ramirez, Sara Fañanas-Baquero, Virginia Nieto-Romero, Andrea Molinos-Vicente, Sonia Gutierrez, Eileen Nicoletti, María García-Bravo, Juan A. Bueren, Jonathan D. Schwartz, Jose-Carlos Segovia
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hematopoiesis
gene therapy
lentiviral vectors
erythroid metabolic diseases
pyruvate kinase deficiency
biodistribution
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121001236

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http://www.sciencedirect.com/science/article/pii/S2329050121001236

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