Pharmacological strategies for mutation-independent treatments of Retinitis Pigmentosa VELUX 2019 STRETTOI (DSB.AD004.208)

Area tematica

Scienze biomediche

Area progettuale

Struttura responsabile del progetto di ricerca

Istituto di neuroscienze (IN)

Responsabile di progetto

ENRICA STRETTOI
Telefono: 0503153213
E-mail: strettoi@in.cnr.it

Abstract

RP is an orphan disease, with an incidence of 1:3500 individuals, so far destined to blindness. Enormous genetic variability, with hundreds mutations in different genes, makes gene therapy an unlikely universal approach for this disease. We propose to target widespread biological processes (inflammation and oxidative stress) accompanying photoreceptor degeneration but also contributing to disease worsening, particularly leading to the secondary loss of cones and total vision loss. Delaying cone loss with pharmacological tools will have an enormous impact on the quality of patients' life.
Our project proposes to fill a gap in current knowledge of Retinitis Pigmentosa (RP) by devising a still missing pharmacological treatment which exploits the ability of new molecules to target inflammation and oxidative stress and delay the disease progression. Specifically, we plan to slow down the secondary degeneration of retinal cones, which become exposed to a hostile microenvironment as a consequence of the primary process of rod death. By in vivo drug delivery to RP mice, we will decrease inflammation and oxidative stress, prolonging the lifetime of cones and the time window of useful visio

Obiettivi

Specific aims of this proposal are the followings:
a. To test the hypothesis that inflammation has a pathogenic role in Retinitis Pigmentosa, concurring to the secondary death of cones and loss of daylight vision. Consequently, pharmacological targeting of inflammation can support cone survival and vision rescue
b. To test the hypothesis that natural molecules with known anti-oxidant effects can be employed in vivo to slow down photoreceptor degeneration and limit visual disability in Retinitis Pigmentosa

Data inizio attività

01/03/2019

Parole chiave

retinite pigmentosa, infiammazione, stress ossidativo

Ultimo aggiornamento: 20/05/2024