Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal

Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal

Infantile malignant osteopetrosis is a devastating disorder of early childhood that is frequently fatal and for which there are only limited therapeutic options. Gene therapy utilizing autologous hematopoietic stem and progenitor cells represents a potentially advantageous therapeutic alternative fo...

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Bibliographic Details
Main Authors: Ilana Moscatelli, Elena Almarza, Axel Schambach, David Ricks, Ansgar Schulz, Christopher D. Herzog, Kim Henriksen, Maria Askmyr, Jonathan D. Schwartz, Johan Richter
Format: Article
Language:English
Published: Elsevier 2021-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
infantile malignant osteopetrosis
lentivirus
gene therapy
autosomal recessive osteopetrosis
hematopoietic stem and progenitor cells
osteoclast disorders
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120302576

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http://www.sciencedirect.com/science/article/pii/S2329050120302576

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