"To evaluate whether Saffron supplementation has a beneficial neuro-protective effect for the damaged retinas as consequence of ABCR mutation-STD\/FF. The specific aims of this research are to investigate whether Saffron influences retinal function in STG\/FF patients as well as in an engineered mouse model of STG\/FF with a knock-out of the ABCR gene.. . Methods: We used abcr -\/- mice in balb\/c background and a randomized, double-blind, placebo-controlled, cross-over study (clinicaltrials.gov: NCT01278277). The degree of damage in protected and unprotected retina was assessed by using electrophysiological techniques (electroretinogram, ERG) associated with immunolabeling and morphological analysis (in mice).. Results: The main result obtained is that Saffron treatment is able to prevent retinal neuro-degeneration in ABCR-\/- mice, particularly when the progression of disease was accelerated by environmental factors (constant light exposure). Treatment with Saffron significantly reduces photoreceptor death in ABCR-\/- mice. Results show significant protection of the ERG signal in treated compared to untreated animals. 31 patients with ABCA4-related STD\/FF and a visual acuity ≥ 0.25 were randomly assigned to assume oral Saffron (S, 20 mg) or placebo (P) over a six month period, and then reverted to P or S supplementation for a further six month period. Our clinical results indicate that saffron may prevent progression of central fERG loss in ABCA4-related STD\/FF, supporting antioxidant neuroprotection as an effective treatment strategy in this molecularly-defined retinal degeneration.. . "

Therapeutic strategy targeting photoreceptor oxidative damage in ABCR-related retinal degenerations

BISTI, SILVIA;MACCARONE, RITA;DI PAOLO, MATTIA;ZERTI, DARIN;
2013-01-01

Abstract

"To evaluate whether Saffron supplementation has a beneficial neuro-protective effect for the damaged retinas as consequence of ABCR mutation-STD\/FF. The specific aims of this research are to investigate whether Saffron influences retinal function in STG\/FF patients as well as in an engineered mouse model of STG\/FF with a knock-out of the ABCR gene.. . Methods: We used abcr -\/- mice in balb\/c background and a randomized, double-blind, placebo-controlled, cross-over study (clinicaltrials.gov: NCT01278277). The degree of damage in protected and unprotected retina was assessed by using electrophysiological techniques (electroretinogram, ERG) associated with immunolabeling and morphological analysis (in mice).. Results: The main result obtained is that Saffron treatment is able to prevent retinal neuro-degeneration in ABCR-\/- mice, particularly when the progression of disease was accelerated by environmental factors (constant light exposure). Treatment with Saffron significantly reduces photoreceptor death in ABCR-\/- mice. Results show significant protection of the ERG signal in treated compared to untreated animals. 31 patients with ABCA4-related STD\/FF and a visual acuity ≥ 0.25 were randomly assigned to assume oral Saffron (S, 20 mg) or placebo (P) over a six month period, and then reverted to P or S supplementation for a further six month period. Our clinical results indicate that saffron may prevent progression of central fERG loss in ABCA4-related STD\/FF, supporting antioxidant neuroprotection as an effective treatment strategy in this molecularly-defined retinal degeneration.. . "
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11697/88663
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