Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal
Ilana Moscatelli, Elena Almarza, Axel Schambach, David Ricks, Ansgar Schulz, Christopher D. Herzog, Kim Henriksen, Maria Askmyr, Jonathan D. Schwartz, Johan Richter
Abstract
in autologous hematopoietic stem and progenitor cells.
Topics & Concepts
OsteopetrosisGenetic enhancementMedicineProgenitor cellStem cellDiseaseHematopoietic stem cell transplantationTransplantationImmunologyOncologyPathologyInternal medicineBiologyGeneGeneticsVirus-based gene therapy researchRNA Interference and Gene DeliveryBone Metabolism and Diseases