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Perinatal systemic gene delivery using adeno-associated viral vectorsGene delivery of a mutant TGFβ3 reduces markers of scar tissue formation after cutaneous woundingFunctional characterization of a 13-bp deletion (c.-1522_-1510del13) in the promoter of the von Willebrand factor gene in type 1 von Willebrand disease.AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage.In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters.Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late-gestation nonhuman primates.In utero gene therapy: current challenges and perspectives.Genetic aspects and research development in haemostasis.Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in miceTransduction of fetal mice with a feline lentiviral vector induces liver tumors which exhibit an E2F activation signature.In utero gene transfer to the mouse nervous system.Intra-amniotic delivery of CFTR-expressing adenovirus does not reverse cystic fibrosis phenotype in inbred CFTR-knockout mice.Multiple vitamin K-dependent coagulation zymogens promote adenovirus-mediated gene delivery to hepatocytes.Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector deliveryTargeting of adenovirus serotype 5 (Ad5) and 5/47 pseudotyped vectors in vivo: fundamental involvement of coagulation factors and redundancy of CAR binding by Ad5.In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression.Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.Long-term transgene expression by administration of a lentivirus-based vector to the fetal circulation of immuno-competent mice.Factors influencing adenovirus-mediated airway transduction in fetal mice.Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors.Gene Therapy with Adeno-associated Virus for Cystic Fibrosis.Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system.Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems.Animal models for prenatal gene therapy: rodent models for prenatal gene therapy.
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description
researcher, ORCID id # 0000-0003-1286-1003
@en
wetenschapper
@nl
name
Suzanne M Buckley
@ast
Suzanne M Buckley
@en
Suzanne M Buckley
@es
Suzanne M Buckley
@nl
type
label
Suzanne M Buckley
@ast
Suzanne M Buckley
@en
Suzanne M Buckley
@es
Suzanne M Buckley
@nl
prefLabel
Suzanne M Buckley
@ast
Suzanne M Buckley
@en
Suzanne M Buckley
@es
Suzanne M Buckley
@nl
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P21
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0000-0003-1286-1003