Restoration of normal BMP signaling levels and osteogenic differentiation in FOP mesenchymal progenitor cells by mutant allele-specific targeting

Gene Therapy

Volumn 19, Issue 7, 2012, Pages 786-790

  Kaplan, J ^a,b   Kaplan, F S ^a,b   Shore, E M ^a,b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

ACVR1 ALK2; Allele specific RNAi; bone morphogenetic protein; fibrodysplasia ossificans progressiva; FOP; heterotopic ossification

Indexed keywords

ACTIVIN RECEPTOR LIKE KINASE 1; BONE MORPHOGENETIC PROTEIN RECEPTOR 1A;

ALLELE; ARTICLE; AUTOSOMAL DOMINANT DISORDER; CELL DIFFERENTIATION; FIBRODYSPLASIA OSSIFICANS PROGRESSIVA; GENE MUTATION; HETEROTOPIC OSSIFICATION; HUMAN; HUMAN CELL; MESENCHYMAL STEM CELL; PRIORITY JOURNAL; REGULATORY MECHANISM; SIGNAL TRANSDUCTION;

ACCESSORY NERVE INJURIES; ACTIVIN RECEPTORS, TYPE I; ALLELES; BONE MORPHOGENETIC PROTEIN RECEPTORS, TYPE I; CELL DIFFERENTIATION; CELLS, CULTURED; DENTAL PULP; GENE SILENCING; HUMANS; MESENCHYMAL STROMAL CELLS; MUTATION; MYOSITIS OSSIFICANS; OSTEOGENESIS; RNA, SMALL INTERFERING; SIGNAL TRANSDUCTION;

EID: 84863723222     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2011.152     Document Type: Article

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