Inherited metabolic disorder of the enzyme pyruvate kinase which affects the survival of red blood cells research hub

Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects with PKD

This study is testing a new gene therapy for adults with Pyruvate Kinase Deficiency (PKD). It aims to see if the treatment is safe by using the patient's own modified cells to improve red blood cell health.