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The Ocular Anomalies in a Cystinosis Animal Model Mimic Disease Pathogenesis

• Vasiliki Kalatzis, Nicolas Serratrice, +9 authors Eric J Kremer
• Pediatric Research
• 2007

Cystinosis is a lysosomal storage disorder characterized by abnormal accumulation of cystine, which forms crystals at high concentrations. The causative gene CTNS encodes cystinosin, the lysosomal… (More)

Corneal Transduction by Intra-Stromal Injection of AAV Vectors In Vivo in the Mouse and Ex Vivo in Human Explants

• Claire Hippert, Sandy Ibanes, +4 authors Vasiliki Kalatzis
• PloS one
• 2012

The cornea is a transparent, avascular tissue that acts as the major refractive surface of the eye. Corneal transparency, assured by the inner stroma, is vital for this role. Disruption in stromal… (More)

Corrective GUSB transfer to the canine mucopolysaccharidosis VII cornea using a helper-dependent canine adenovirus vector.

• Nicolas Serratrice, Aurélie Cubizolle, +11 authors Eric J Kremer
• Journal of controlled release : official journal…
• 2014

Corneal transparency is maintained, in part, by specialized fibroblasts called keratocytes, which reside in the fibrous lamellae of the stroma. Corneal clouding, a condition that impairs visual… (More)

Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.

• Aurélie Cubizolle, Nicolas Serratrice, +14 authors Eric J Kremer
• Molecular therapy : the journal of the American…
• 2014

Severe deficiency in lysosomal β-glucuronidase (β-glu) enzymatic activity results in mucopolysaccharidosis (MPS) VII, an orphan disease with symptoms often appearing in early childhood. Symptoms are… (More)

Cystine accumulation in the CNS results in severe age-related memory deficits.

• Tangui Maurice, Claire Hippert, +5 authors Vasiliki Kalatzis
• Neurobiology of aging
• 2009

Cystinosis is a lysosomal storage disorder characterised by progressive cystine accumulation. The causative gene, CTNS, encodes cystinosin, the lysosomal cystine transporter. Neurological… (More)

Central nervous system delivery of helper-dependent canine adenovirus corrects neuropathology and behavior in mucopolysaccharidosis type VII mice.

• Lorena Ariza, Lydia Giménez-Llort, +8 authors Assumpció Bosch
• Human gene therapy
• 2014

Canine adenovirus type 2 vectors (CAV-2) are promising tools to treat global central nervous system (CNS) disorders because of their preferential transduction of neurons and efficient retrograde… (More)

Exogenous LRRK2G2019S induces parkinsonian-like pathology in a nonhuman primate.

• Nadine Mestre-Francés, Nicolas Serratrice, +15 authors Eric J Kremer
• JCI insight
• 2018

Parkinson's disease (PD) is the second most prevalent neurodegenerative disease among the elderly. To understand its pathogenesis and to test therapies, animal models that faithfully reproduce key… (More)

Contrasting effects of human, canine, and hybrid adenovirus vectors on the phenotypical and functional maturation of human dendritic cells: implications for clinical efficacy.

• Matthieu Perreau, Franck J D Mennechet, +4 authors Eric J Kremer
• Journal of virology
• 2007

Antipathogen immune responses create a balance between immunity, tolerance, and immune evasion. However, during gene therapy most viral vectors are delivered in substantial doses and are incapable of… (More)

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