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

MeSH terms

• Anemia, Hemolytic, Congenital Nonspherocytic* / enzymology
• Anemia, Hemolytic, Congenital Nonspherocytic* / genetics
• Anemia, Hemolytic, Congenital Nonspherocytic* / therapy
• Clinical Trials as Topic
• Genetic Therapy*
• Humans
• Pyruvate Kinase / deficiency*
• Pyruvate Kinase / genetics
• Pyruvate Metabolism, Inborn Errors* / enzymology
• Pyruvate Metabolism, Inborn Errors* / genetics
• Pyruvate Metabolism, Inborn Errors* / therapy