DS0411 - Recherche translationnelle en santé

Towards a comprehension of transferrin protective effects to its therapeutic application in retinal degeneration – Transfiron

Submission summary

Dysregulation of local ocular iron metabolism leading to iron excess is now considered as important contributor to the pathogenesis of neurodegeneration diseases. Age-related macular degeneration (AMD) is the most common cause of irreversible vision loss in the elderly worldwide, involving both genetic and environmental factors. Oxidative stress and inflammation are implicated in the pathology of the two forms (dry and wet) of advanced AMD. Photoreceptors (PRs) are extremely sensitive to iron, that accumulates in the retina of patients with AMD. Iron excess is therefore a potential target for neuroprotection in AMD. But no animal model recapitulates AMD because rodents do not have a macula. In retinal detachment (RD) oxidative stress induces a rapid degeneration of the retina. Animal models are relevant and RD is optimal for first clinical proof of concept.
Transfiron project aims at:
1) Study iron metabolism deregulation leading to iron accumulation in the ocular fluids of patients, with AMD and RD according to their phenotype as compared to coµuyètr’ntrol patients.
2) Screen for the optimal Tf neuroprotective effect in vitro: The influence of post-translational Tf modifications, Tf-proteins partners and iron-related proprieties on its neuroprotective effect will be studied in vitro according to molecular and biochemical tools.
3) Test and select optimized Tf variants for clinical use. Tf mutants will be screened for increased neuroprotective efficiency in retinal explants and improved stability and biodisponibility on relevant animal models in vivo.

This project aims at: - completing our knowledge on iron metabolism in eyes with retinal degenerative diseases, - characterizing and optimizing the neuroprotective activity of Tf for future clinical applications in clinical diseases (RD and AMD).

Project coordination

Emilie PICARD (INSERM UMRS1138 team17)

The author of this summary is the project coordinator, who is responsible for the content of this summary. The ANR declines any responsibility as for its contents.


UMRS1138 INSERM UMRS1138 team17

Help of the ANR 388,024 euros
Beginning and duration of the scientific project: September 2015 - 36 Months

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