Dany Perocheau

Great Ormond Street Institute of Child Health, University College London (UCL)

Affiliated withUniversity College London (UCL)

Research Area

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JoVE Journal Publications

ArticleTotal : 1	Year
Publication title	2024

Other Publications

Article	Year
Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy. American journal of obstetrics and gynecology\| PubMed ID: 16157120	2005
Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector delivery. Journal of immunology research\| PubMed ID: 25759840	2015
In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters. Scientific reports\| PubMed ID: 26138224	2015
Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice. Scientific reports\| PubMed ID: 28157201	2017
Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 28550974	2017
Continual conscious bioluminescent imaging in freely moving somatotransgenic mice. Scientific reports\| PubMed ID: 28743959	2017
Fetal gene therapy for neurodegenerative disease of infants. Nature medicine\| PubMed ID: 30013199	2018
Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort. Human gene therapy\| PubMed ID: 30027761	2019
Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice. The American journal of pathology\| PubMed ID: 30036519	2018
Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer. Nature communications\| PubMed ID: 30158522	2018
Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload. Cell death and differentiation\| PubMed ID: 31685979	2020
Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors. Scientific reports\| PubMed ID: 32034258	2020
Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth. Frontiers in immunology\| PubMed ID: 32117260	2020
Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload. Cell death and differentiation\| PubMed ID: 32152554	2020
An Whole-Organ Liver Engineering for Testing of Genetic Therapies. iScience\| PubMed ID: 33305175	2020
Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation. Molecular therapy. Methods & clinical development\| PubMed ID: 33553484	2021
Gene therapy for inherited metabolic diseases. Journal of mother and child\| PubMed ID: 33554501	2020
Clinical applications for exosomes: Are we there yet? British journal of pharmacology\| PubMed ID: 33751579	2021
The exosome journey: from biogenesis to uptake and intracellular signalling. Cell communication and signaling : CCS\| PubMed ID: 33892745	2021
Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism. Science translational medicine\| PubMed ID: 34011628	2021
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys. Molecular therapy. Methods & clinical development\| PubMed ID: 34703837	2021
Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors. Human gene therapy\| PubMed ID: 36927149	2023
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria. Journal of inherited metabolic disease\| PubMed ID: 38044746	2023
mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria. Science translational medicine\| PubMed ID: 38198573	2024
precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases. F1000Research\| PubMed ID: 38618017	2023