Generation of gene-corrected functional osteoclasts from osteopetrotic induced pluripotent stem cells

Hematopoietic Stem Cell-Targeted Neonatal Gene Therapy with a Clinically Applicable Lentiviral Vector Corrects Osteopetrosis in oc/oc Mice

(2019) Henrik Löfvall et al.   HUMAN GENE THERAPY

Osteopetrotic induced pluripotent stem cells derived from patients with different disease-associated mutations by non-integrating reprogramming methods

(2019) Fatma Visal Okur et al.   Stem Cell Research & Therapy

TCIRG1 Transgenic Rescue of Osteoclast Function Using Induced Pluripotent Stem Cells Derived from Patients with Infantile Malignant Autosomal Recessive Osteopetrosis

(2019) Weili Chen et al.   JOURNAL OF BONE AND JOINT SURGERY-AMERICAN VOLUME

Generation of 3 clones of induced pluripotent stem cells (iPSCs) from a patient affected by Autosomal Recessive Osteopetrosis due to mutations in TCIRG1 gene.

(2019) Gaetana Lanzi et al.   Stem Cell Research

Successful hematopoietic stem cell transplantation for osteopetrosis using reduced intensity conditioning

(2018) Bella Shadur et al.   PEDIATRIC BLOOD & CANCER

Targeting NSG Mice Engrafting Cells with a Clinically Applicable Lentiviral Vector Corrects Osteoclasts in Infantile Malignant Osteopetrosis

(2017) Ilana Moscatelli et al.   HUMAN GENE THERAPY

Diagnosis and Management of Osteopetrosis: Consensus Guidelines From the Osteopetrosis Working Group

(2017) Calvin C Wu et al.   JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM

Ex vivo Generation of Genetically Modified Macrophages from Human Induced Pluripotent Stem Cells

(2017) Mania Ackermann et al.   TRANSFUSION MEDICINE AND HEMOTHERAPY

Generation of integration free induced pluripotent stem cells from fibrodysplasia ossificans progressiva (FOP) patients from urine samples

(2016) Laura Hildebrand et al.   Stem Cell Research

A minimal ubiquitous chromatin opening element (UCOE) effectively prevents silencing of juxtaposed heterologous promoters by epigenetic remodeling in multipotent and pluripotent stem cells

(2015) Uta Müller-Kuller et al.   NUCLEIC ACIDS RESEARCH

Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts

(2015) Tui Neri et al.   Stem Cell Reports

Osteopetrosis: genetics, treatment and new insights into osteoclast function

(2013) Cristina Sobacchi et al.   Nature Reviews Endocrinology

Efficient, Long Term Production of Monocyte-Derived Macrophages from Human Pluripotent Stem Cells under Partly-Defined and Fully-Defined Conditions

(2013) Bonnie van Wilgenburg et al.   PLoS One

Nonablative neonatal bone marrow transplantation rapidly reverses severe murine osteopetrosis despite low-level engraftment and lack of selective expansion of the osteoclastic lineage

(2010) Carmen Flores et al.   JOURNAL OF BONE AND MINERAL RESEARCH

Prospects for Gene Therapy of Osteopetrosis

(2009) Maria Askmyr et al.   CURRENT GENE THERAPY

Committed osteoclast precursors colonize the bone and improve the phenotype of a mouse model of autosomal recessive osteopetrosis

(2009) Alfredo Cappariello et al.   JOURNAL OF BONE AND MINERAL RESEARCH

Genetics, pathogenesis and complications of osteopetrosis

(2007) Andrea Del Fattore et al.   BONE