Article
Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis.
Human gene therapy - 1 Aug 2018
Moscatelli Ilana, Löfvall Henrik, Schneider Thudium Christian, Rothe Michael, Montano Carmen, Kertész Zsuzsanna, Sirin Mehtap, Schulz Ansgar, Schambach Axel, Henriksen Kim, Richter Johan
Abstract excerpt
Infantile malignant osteopetrosis (IMO) is a rare, lethal, autosomal recessive disorder characterized by nonfunctional osteoclasts. More than 50% of the patients have mutations in the TCIRG1 gene, encoding for a subunit of the osteoclast proton pump. The aim of this study was to develop a clinically applicable lentiviral vector expressing TCIRG1 to correct osteoclast function in IMO. Two mammalian promoters were...
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