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      Trial Interventions in ABCA4- Retinopathy

      Preprint
      In review
      research-article
        1 ,
      ScienceOpen Preprints
      ScienceOpen
      ABCA4, retinopathy , pathophysiology, ABCA4 transporter physiology
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            Abstract

            Abstract: Inherited retinal diseases collectively are one of the leading causes of visual impairment worldwide. ABCA4 retinopathy is the most common inherited retinal disease. In the last few years, there are many advances in the understanding of this disease, which led to many interventional trials with promising results at the moment. This paper is going to present a brief background up to date knowledge in the context of the disease pathophysiology, the main mechanisms involved in this disease and related diseases, and different approaches of the intervention trials. I will be using a literature review to highlight the main structural, physiological, chemical, functional characteristics of ABCA4 transporter physiology. This will be followed by a brief discussion about pathophysiology. Next, analysing, extracting and comparing different interventional options and presenting the current clinical trials for each will be done. This paper expected to discusses and rationally compare between different treatment trials approaches their advantages over others and challenges. A sensible, possible application from current trials from different diseases with similar pathogenesis and possible research opportunities will be discussed.

            Content

            Author and article information

            Journal
            ScienceOpen Preprints
            ScienceOpen
            5 June 2020
            Affiliations
            [1 ] University College London
            Article
            10.14293/S2199-1006.1.SOR-.PPZ0NMP.v1
            93417753-23f2-4108-9367-22f47ac841e5

            This work has been published open access under Creative Commons Attribution License CC BY 4.0 , which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Conditions, terms of use and publishing policy can be found at www.scienceopen.com .


            Data sharing not applicable to this article as no datasets were generated or analysed during the current study.
            Medicine
            ABCA4,retinopathy ,pathophysiology,ABCA4 transporter physiology

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