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Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism.
2023
Francesca Tucci, and Giulia Consiglieri, and Matilde Cossutta, and Maria Ester Bernardo
Pediatric Immunohematology and Bone Marrow Transplantation, IRCCS San Raffaele Scientific Institute, Milan, Italy.
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Francesca Tucci, and Giulia Consiglieri, and Matilde Cossutta, and Maria Ester Bernardo
Haematopoietic stem cell gene therapy in inborn errors of metabolism.
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Gene therapy for inborn errors of liver metabolism.
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