Dany Perocheau

Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy.

Evidence for contribution of CD4+ CD25+ regulatory T cells in maintaining immune tolerance to human factor IX following perinatal adenovirus vector delivery.

In vivo bioimaging with tissue-specific transcription factor activated luciferase reporters.

Longitudinal in vivo bioimaging of hepatocyte transcription factor activity following cholestatic liver injury in mice.

Eliminating HIV-1 Packaging Sequences from Lentiviral Vector Proviruses Enhances Safety and Expedites Gene Transfer for Gene Therapy.

Continual conscious bioluminescent imaging in freely moving somatotransgenic mice.

Fetal gene therapy for neurodegenerative disease of infants.

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort.

Ascending Vaginal Infection Using Bioluminescent Bacteria Evokes Intrauterine Inflammation, Preterm Birth, and Neonatal Brain Injury in Pregnant Mice.

Argininosuccinic aciduria fosters neuronal nitrosative stress reversed by Asl gene transfer.

Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload.

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors.

Cervical Gene Delivery of the Antimicrobial Peptide, Human β-Defensin (HBD)-3, in a Mouse Model of Ascending Infection-Related Preterm Birth.

Correction: Impaired cellular bioenergetics caused by GBA1 depletion sensitizes neurons to calcium overload.

An Whole-Organ Liver Engineering for Testing of Genetic Therapies.

Re-structuring lentiviral vectors to express genomic RNA via cap-dependent translation.

Gene therapy for inherited metabolic diseases.

Clinical applications for exosomes: Are we there yet?

The exosome journey: from biogenesis to uptake and intracellular signalling.

Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism.

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.

Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors.

The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria.

mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria.

precision-cut liver slices model disease phenotype and monitor therapeutic response for liver monogenic diseases.