Experimental studies on cell and gene therapies for retinal dystrophies with a particular focus on ABCA4 retinopathies

Aneta Ścieżyńska; Dominika Oziębło; Monika Ołdak

doi:10.5114/ko.2016.71660

POLSKI

eISSN: 2719-3209
ISSN: 0023-2157

Klinika Oczna / Acta Ophthalmologica Polonica

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1/2016
vol. 118

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abstract:

Review paper

Experimental studies on cell and gene therapies for retinal dystrophies with a particular focus on ABCA4 retinopathies

Aneta Ścieżyńska

¹

,

Dominika Oziębło

²

,

Monika Ołdak

^{1, 2, 3}

Katedra i Zakład Histologii i Embriologii, Centrum Biostruktury, Warszawski Uniwersytet Medyczny
Zakład Genetyki, Instytut Fizjologii i Patologii Słuchu w Warszawie
Samodzielny Publiczny Kliniczny Szpital Okulistyczny w Warszawie

DOI: https://doi.org/10.5114/ko.2016.71660

Online publish date: 2017/11/29

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Retinal dystrophies lead to gradual irreversible vision deterioration. The ABCA4 retinopathies constitute an important group of retinal dystrophies. However, there are no effective therapies available for this group of diseases. Yet, with the advent of molecular therapies, the development of prospective therapeutic approaches seems feasible. The paper summarizes recent advances in gene and cell therapy that may be implemented in retinal dystrophies, especially in ABCA4-associated diseases.

keywords:

Experimental studies on cell and gene therapies for retinal dystrophies with a particular focus on ABCA4 retinopathies

Aneta Ścieżyńska 1 , Dominika Oziębło 2 , Monika Ołdak 1, 2, 3

ABCA4 gene, Stargardt disease, retinal dystrophies, gene therapy, cell therapy, clinical trials

Aneta Ścieżyńska

¹

,

Dominika Oziębło

²

,

Monika Ołdak

^{1, 2, 3}