Gene Therapy in Inherited Retinal Diseases: An Update on Current State of the Art.
Gene Therapy in Inherited Retinal Diseases: An Update on Current State of the Art.
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DOI：
10.3389/fmed.2021.750586
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发表时间：
2021
影响因子：
3.9
通讯作者：
Battaglia Parodi M
中科院分区：
文献类型：
--
作者：
Amato A;Arrigo A;Aragona E;Manitto MP;Saladino A;Bandello F;Battaglia Parodi M
背景：基因治疗在视网膜疾病领域还不能被认为是一个遥远的前景，但却是一个切实可行的治疗选择。虽然仍然局限于实验环境中，初步结果是有希望的，并提供了一个整体的情况下，表明我们是不是远离基因治疗在临床环境中的应用。本综述的主要目的是提供一个完整的和最新的概述，目前的最先进的基因治疗应用于视网膜疾病和未来的前景。研究方法：我们仔细修订了整个文献，报告了所有与实验程序相关的发现，以及基因治疗在视网膜疾病中的应用前景。还报告了每种视网膜疾病的临床背景和遗传特征的详细描述。结果：目前的文献强烈支持视网膜疾病的基因治疗方案的希望。尽管基因治疗在某些视网膜疾病如无脉络膜症（CHM）、色素性视网膜炎（RP）和Leber先天性黑蒙（LCA）的研究中处于后期阶段，但在其他视网膜疾病的临床实践中，基因治疗仍然相当遥远。结论：基因治疗是一种非常有前途的视网膜疾病治疗手段。在这篇综述中报道的实验数据提供了一个强烈的希望，基因治疗将有效地在临床实践中，在未来几年。
Background: Gene therapy cannot be yet considered a far perspective, but a tangible therapeutic option in the field of retinal diseases. Although still confined in experimental settings, the preliminary results are promising and provide an overall scenario suggesting that we are not so far from the application of gene therapy in clinical settings. The main aim of this review is to provide a complete and updated overview of the current state of the art and of the future perspectives of gene therapy applied on retinal diseases. Methods: We carefully revised the entire literature to report all the relevant findings related to the experimental procedures and the future scenarios of gene therapy applied in retinal diseases. A clinical background and a detailed description of the genetic features of each retinal disease included are also reported. Results: The current literature strongly support the hope of gene therapy options developed for retinal diseases. Although being considered in advanced stages of investigation for some retinal diseases, such as choroideremia (CHM), retinitis pigmentosa (RP), and Leber's congenital amaurosis (LCA), gene therapy is still quite far from a tangible application in clinical practice for other retinal diseases. Conclusions: Gene therapy is an extremely promising therapeutic tool for retinal diseases. The experimental data reported in this review offer a strong hope that gene therapy will be effectively available in clinical practice in the next years.
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DOI：
10.1038/mt.2015.220
发表时间：
2016-03
期刊：
Molecular therapy : the journal of the American Society of Gene Therapy
影响因子：
--
作者：
Bakondi B;Lv W;Lu B;Jones MK;Tsai Y;Kim KJ;Levy R;Akhtar AA;Breunig JJ;Svendsen CN;Wang S
通讯作者：
Wang S
DOI：
10.1097/00006982-200507000-00012
发表时间：
2005-07-01
影响因子：
3.3
作者：
Apushkin, MA;Fishman, GA;Rajagopalan, AS
通讯作者：
Rajagopalan, AS
影响因子：
3
作者：
Arrigo, Alessandro;Romano, Francesco;Parodi, Maurizio Battaglia
通讯作者：
Parodi, Maurizio Battaglia
DOI：
10.3390/medicina57010064
发表时间：
2021-01-12
期刊：
Medicina (Kaunas, Lithuania)
影响因子：
--
作者：
Abbouda A;Avogaro F;Moosajee M;Vingolo EM
通讯作者：
Vingolo EM
影响因子：
9.8
作者：
Albert, Silvia;Garanto, Alejandro;Cremers, Frans P. M.
通讯作者：
Cremers, Frans P. M.