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Literature DB >> 30040511

Gene therapy in inherited retinal degenerative diseases, a review.

Vitor K L Takahashi^1,2,3, Júlia T Takiuti^1,2,4, Ruben Jauregui^1,2,5, Stephen H Tsang^1,2,6.

Abstract

Hereditary diseases of the retina represent a group of diseases with several heterogeneous mutations that have the common end result of progressive photoreceptor death leading to blindness. Retinal degenerations encompass multifactorial diseases such as age-related macular degeneration, Leber congenital amaurosis, Stargardt disease, and retinitis pigmentosa. Although there is currently no cure for degenerative retinal diseases, ophthalmology has been at the forefront of the development of gene therapy, which offers hope for the treatment of these conditions. This article will explore an overview of the clinical trials of gene supplementation therapy for retinal diseases that are underway or planned for the near future.

Entities: Disease

Keywords: Adeno-associated virus; gene therapy; inherited retinal diseases

Mesh：

Year: 2018 PMID： 30040511 DOI： 10.1080/13816810.2018.1495745

Source DB: PubMed Journal: Ophthalmic Genet ISSN： 1381-6810 Impact factor: 1.803

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Cited

24 in total

Review 1. Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.

Authors: Galaxy Y Cho; Kyle Bolo; Karen Sophia Park; Jesse D Sengillo; Stephen H Tsang
Journal: Mol Diagn Ther Date: 2019-02 Impact factor: 4.074

2. Spectrum of Disease Severity and Phenotype in Choroideremia Carriers.

Authors: Ruben Jauregui; Karen Sophia Park; Akemi J Tanaka; Ahra Cho; Maarjaliis Paavo; Jana Zernant; Jasmine H Francis; Rando Allikmets; Janet R Sparrow; Stephen H Tsang
Journal: Am J Ophthalmol Date: 2019-06-08 Impact factor: 5.258

3. Choriocapillaris flow loss in center-involving retinitis pigmentosa: a quantitative optical coherence tomography angiography study using a novel classification system.

Authors: Sally S Ong; T Y Alvin Liu; Ximin Li; Mandeep S Singh
Journal: Graefes Arch Clin Exp Ophthalmol Date: 2021-05-31 Impact factor: 3.117

Review 4. Stem cell transplantation as a progressing treatment for retinitis pigmentosa.

Authors: Sedighe Hosseini Shabanan; Homa Seyedmirzaei; Alona Barnea; Sara Hanaei; Nima Rezaei
Journal: Cell Tissue Res Date: 2022-01-10 Impact factor: 5.249

5. Targeted next-generation sequencing identifies ABCA4 mutations in Chinese families with childhood-onset and adult-onset Stargardt disease.

Authors: Ling-Hui Qu; Xin Jin; Chao Zeng; Nian-Gou Zhou; Yan-Hong Liu; Ye Lin
Journal: Biosci Rep Date: 2021-06-25 Impact factor: 3.840

Gene therapy in inherited retinal degenerative diseases, a review.

Review 1. Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.

2. Spectrum of Disease Severity and Phenotype in Choroideremia Carriers.

3. Choriocapillaris flow loss in center-involving retinitis pigmentosa: a quantitative optical coherence tomography angiography study using a novel classification system.

Review 4. Stem cell transplantation as a progressing treatment for retinitis pigmentosa.

5. Targeted next-generation sequencing identifies ABCA4 mutations in Chinese families with childhood-onset and adult-onset Stargardt disease.

Review 6. Impact of Photoreceptor Loss on Retinal Circuitry.

Review 7. CRISPR-Based Genome Editing as a New Therapeutic Tool in Retinal Diseases.

8. Differences in Intraretinal Pigment Migration Across Inherited Retinal Dystrophies.

9. PKM2 ablation enhanced retinal function and survival in a preclinical model of retinitis pigmentosa.

10. Risk Stratification and Clinical Utility of Polygenic Risk Scores in Ophthalmology.