Julien Baruteau

Transcatheter closure of patent ductus arteriosus: past, present and future.

Perinatal systemic gene delivery using adeno-associated viral vectors.

Expanding the phenotype in argininosuccinic aciduria: need for new therapies.

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects.

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.

Foamy Virus Vectors Transduce Visceral Organs and Hippocampal Structures following In Vivo Delivery to Neonatal Mice.

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

Fetal gene therapy for neurodegenerative lysosomal storage diseases.

Liver neoplasms in methylmalonic aciduria: An emerging complication.

Urea Cycle Related Amino Acids Measured in Dried Bloodspots Enable Long-Term In Vivo Monitoring and Therapeutic Adjustment.

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.

Clinical and Molecular Features of Early Infantile Niemann Pick Type C Disease.

Novel therapies for mucopolysaccharidosis type III.

cGAS-mediated induction of type I interferon due to inborn errors of histone pre-mRNA processing.

Gene therapy for inherited metabolic diseases.

Disentangling molecular and clinical stratification patterns in beta-galactosidase deficiency.

Clinical applications for exosomes: Are we there yet?

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

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development.

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

ASL expression in ALDH1A1 neurons in the substantia nigra metabolically contributes to neurodegenerative phenotype.

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

Modelling urea cycle disorders using iPSCs.

Inherited and acquired vitamin B12 deficiencies: Which administration route to choose for supplementation?

Three-Country Snapshot of Ornithine Transcarbamylase Deficiency.

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

Natural history of epilepsy in argininosuccinic aciduria provides new insights into pathophysiology: A retrospective international study.

Gene therapy for urea cycle defects: An update from historical perspectives to future prospects.

Recapitulation of Skewed X-Inactivation in Female Ornithine Transcarbamylase-Deficient Primary Human Hepatocytes in the FRG Mouse: A Novel System for Developing Epigenetic Therapies.

Prolonged respiratory failure responds to conventional therapy in isolated homocysteine remethylation defects.

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

Liver transplantation in ornithine transcarbamylase deficiency: A retrospective multicentre cohort study.

Liver-directed gene therapy for inherited metabolic diseases.

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

Mission possible: Gene therapy for inherited metabolic diseases.

Generation of induced pluripotent stem cells (UCLi024-A) from a patient with argininosuccinate lyase deficiency carrying a homozygous c.437G > A (p.Arg146Gln) mutation.

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

Asymptomatic pediatric presentation of S-adenosylhomocysteine hydrolase deficiency.