L. Vérot

发表

Improved Behavior and Neuropathology in the Mouse Model of Sanfilippo Type IIIB Disease after Adeno-Associated Virus-Mediated Gene Transfer in the Striatum

A. Cressant, R. Froissart, M. Vanier, 2004, The Journal of Neuroscience.

Niemann–Pick C disease: functional characterization of three NPC2 mutations and clinical and molecular update on patients with NPC2

G. Millat, M. Vanier, M. Vanier, 2007, Clinical genetics.

Prevention of neuropathology in the mouse model of hurler syndrome

C. Caillaud, M. Vanier, J. Heard, 2004, Annals of neurology.

Partial cure of established disease in an animal model of metachromatic leukodystrophy after intracerebral adeno-associated virus-mediated gene transfer

A. Benraiss, G. Nagels, V. Gieselmann, 2007, Gene Therapy.

Intracerebral adeno-associated virus-mediated gene transfer in rapidly progressive forms of metachromatic leukodystrophy.

A. Benraiss, G. Nagels, M. Vidaud, 2006, Human molecular genetics.

Niemann–Pick type C disease involves disrupted neurosteroidogenesis and responds to allopregnanolone

S. Mellon, Wenhui Gong, L. D. Griffin, 2004, Nature Medicine.

Gene therapy of the brain in the dog model of Hurler's syndrome

S. Raoul, B. Schwartz, N. Ferry, 2006, Annals of neurology.

Bone marrow transplantation for feline mucopolysaccharidosis I.

C. Vite, G. Aguirre, S. Evans, 2007, Molecular genetics and metabolism.

Genetic evidence for nonredundant functional cooperativity between NPC1 and NPC2 in lipid transport.

M. Shen, S. Walkley, D. Sleat, 2004, Proceedings of the National Academy of Sciences of the United States of America.

[Fatal neonatal respiratory distress in Niemann-Pick C2 and prenatal diagnosis with mutations in gene HE1/NPC2].

F. Gottrand, G. Millat, M. Vanier, 2005, Archives de pediatrie : organe officiel de la Societe francaise de pediatrie.

Localization of a non‐syndromic X‐linked mental retardation gene (MRX80) to Xq22‐q24

R. Touraine, H. Plauchu, P. Edery, 2003, American journal of medical genetics. Part A.