about
Facioscapulohumeral dystrophy: incomplete suppression of a retrotransposed geneA unifying genetic model for facioscapulohumeral muscular dystrophyEndogenous DUX4 expression in FSHD myotubes is sufficient to cause cell death and disrupts RNA splicing and cell migration pathwaysRNA transcripts, miRNA-sized fragments and proteins produced from D4Z4 units: new candidates for the pathophysiology of facioscapulohumeral dystrophyDigenic inheritance of an SMCHD1 mutation and an FSHD-permissive D4Z4 allele causes facioscapulohumeral muscular dystrophy type 2Asymmetric bidirectional transcription from the FSHD-causing D4Z4 array modulates DUX4 production.Foamy virus vector integration sites in normal human cellsSystemic delivery of genes to striated muscles using adeno-associated viral vectors.Inter-individual differences in CpG methylation at D4Z4 correlate with clinical variability in FSHD1 and FSHD2.VISA--Vector Integration Site Analysis server: a web-based server to rapidly identify retroviral integration sites from next-generation sequencing.Chromatin structure of two genomic sites for targeted transgene integration in induced pluripotent stem cells and hematopoietic stem cells.A cross sectional study of two independent cohorts identifies serum biomarkers for facioscapulohumeral muscular dystrophy (FSHD).Wnt/β-catenin signaling suppresses DUX4 expression and prevents apoptosis of FSHD muscle cells.A Human Pluripotent Stem Cell Model of Facioscapulohumeral Muscular Dystrophy-Affected Skeletal Muscles.Frequent endonuclease cleavage at off-target locations in vivo.Adeno-associated virus vectors integrate at chromosome breakage sites.Efficient PRNP gene targeting in bovine fibroblasts by adeno-associated virus vectors.Expression patterns of FSHD-causing DUX4 and myogenic transcription factors PAX3 and PAX7 are spatially distinct in differentiating human stem cell culturesEfficient KRT14 targeting and functional characterization of transplanted human keratinocytes for the treatment of epidermolysis bullosa simplex.Integration bias of gammaretrovirus vectors following transduction and growth of primary mouse hematopoietic progenitor cells with and without selection.Unique integration profiles in a canine model of long-term repopulating cells transduced with gammaretrovirus, lentivirus, or foamy virus.Gene targeting in vivo by adeno-associated virus vectors.Chromosomal effects of adeno-associated virus vector integration.Comparison of HIV-derived lentiviral and MLV-based gammaretroviral vector integration sites in primate repopulating cells.AAV vector integration sites in mouse hepatocellular carcinoma.Underperforming Big Ideas in Biomedical Research.Gene therapy for hemophiliaAAV-mediated gene targetingLongitudinal features of STIR bright signal in FSHD
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description
hulumtues
@sq
researcher
@en
wetenschapper
@nl
հետազոտող
@hy
name
Daniel G Miller
@es
Daniel G Miller
@fr
Daniel G Miller
@sl
Daniel G. Miller
@en
Daniel G. Miller
@nl
type
label
Daniel G Miller
@es
Daniel G Miller
@fr
Daniel G Miller
@sl
Daniel G. Miller
@en
Daniel G. Miller
@nl
prefLabel
Daniel G Miller
@es
Daniel G Miller
@fr
Daniel G Miller
@sl
Daniel G. Miller
@en
Daniel G. Miller
@nl
P106
P1153
54950430300
P21
P31
P496
0000-0002-5700-3048