Antoine Gardin

Modern therapeutic approaches to liver-related disorders.

E. Andersson, P. Strnad, E. Gonzales, 2022, Journal of hepatology.

Long term follow-up after haematopoietic stem cell transplantation for mucopolysaccharidosis type I-H: a retrospective study of 51 patients

N. Boddaert, S. Pannier, D. Lacombe, 2022, Bone Marrow Transplantation.

Maternal and Offspring Pools of Osteocalcin Influence Brain Development and Functions

J. Mann, T. Horvath, T. Langer, 2013, Cell.

A retrospective multicentric study on 34 patients with Niemann-Pick type C disease and early liver involvement in France.

A. Brassier, M. Schiff, B. Héron, 2022, The Journal of pediatrics.

Pathological modeling of glycogen storage disease type III with CRISPR/Cas9 edited human pluripotent stem cells

L. Hoch, Manon Benabides, X. Nissan, 2023, Frontiers in Cell and Developmental Biology.

JAG1 and THBS2 Mutations in a Child Presenting With Incomplete Alagille Syndrome

E. Jacquemin, J. Bouligand, M. Almes, 2023, JPGN Reports.

Outcomes of 38 patients with PFIC3: Impact of genotype and of response to ursodeoxycholic acid therapy

B. Bénichou, E. Jacquemin, O. Ackermann, 2023, JHEP Reports.

Current limitations of gene therapy for rare pediatric diseases: Lessons learned from clinical experience with AAV vectors.

G. Ronzitti, Antoine Gardin, 2023, Archives de pediatrie : organe officiel de la Societe francaise de pediatrie.

A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III

L. Hoch, G. Ronzitti, J. Esque, 2023, The Journal of clinical investigation.