Abstract
1/2016
vol. 118
Review paper
Experimental studies on cell and gene therapies for retinal dystrophies with a particular focus on ABCA4 retinopathies
- 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
Online publish date: 2017/11/29
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
ABCA4 gene, Stargardt disease, retinal dystrophies, gene therapy, cell therapy, clinical trials
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