Who should be eligible for gene therapy clinical trials in red blood cell pyruvate kinase deficiency (PKD)?: Toward an expanded definition of severe PKD

Bibliographic Details
Main Authors:	Barcellini, W. (Author), Bianchi, P. (Author), Glader, B. (Author), Grace, R.F (Author), López Lorenzo, J.L (Author), Navarro Ordoñez, S. (Author), Nicoletti, E. (Author), Schwartz, J.D (Author), Segovia, J.C (Author), Sevilla, J. (Author), Shah, A.J (Author), Zanella, A. (Author)
Format:	Article
Language:	English
Published:	John Wiley and Sons Inc 2022
Subjects:	adenosine triphosphate allogeneic hematopoietic stem cell transplantation anemia Anemia, Hemolytic, Congenital Nonspherocytic attitude to illness bilirubin blood transfusion CD34 antigen clinical effectiveness clinical feature clinical trial (topic) Clinical Trials as Topic diagnostic procedure disease burden disease classification disease severity disorders of carboxylic acid metabolism enzymology erythrocyte disorder erythropoietin fatigue follow up gene gene mutation gene therapy Genetic Therapy genetic variability genetics genotype phenotype correlation hemoglobin hemolytic anemia hereditary hemolytic anemia human Humans iron chelation iron overload Letter mitapivat molecular pathology nonsense mutation patient care PKLR gene pyruvate kinase Pyruvate Kinase pyruvate kinase deficiency Pyruvate Metabolism, Inborn Errors reticulocyte risk assessment RNA splice site safety splenectomy survival analysis toxicity testing
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