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Gene Therapy Development for Vision Disorder

Vision disorder, different from an eye disease, is not only caused by the eye injury but also as a result of the dysfunction at other locations in the optic pathway. The disease-causing genes have been identified in some inherited vision disorder, which greatly facilitates the potential for gene therapy. Gene therapies have a promising future in the treatment of vision disorder and have been used in inherited retinal and optic nerve degenerations, such as the Leber’s congenital amaurosis (LCA), choroideremia (CHM), retinitis pigmentosa (RP), and Leber’s hereditary optic neuropathy (LHON), etc. Creative Biolabs is committed to providing the high-quality custom service in the field of gene therapy development. Our well-trained technical and advanced platform can offer the most appropriate research plan to best suit your program and budget requirements.

Intravitreal and subretinal injection of gene therapeutic viral vectors Figure 1. Intravitreal and subretinal injection of gene therapeutic viral vectors (Moore 2017)

Gene Therapy Development Services for Vision Disorder

LCA is a visually devastating disease and belongs to the heterogenous group of infantile-onset, retinal dystrophy caused by a variety of genetic mutations. The RPE65 gene-encoding retinoid isomerohydrolase is one of the pathologic genetic mutations of interest in LCA. Gene-replacement therapy such as the subretinal delivery of recombinant AAV vectors containing RPE65 coding sequence has been shown to enhance the electrophysiological responses and functional vision in RPE65 mutated animal model. In addition to RPE65, the mutation in guanylate cyclase 2D (GUCY2D) and the retinitis pigmentosa GTPase regulator protein (RPGRIP1) gene are implicated in LCA development. AAV vector-mediated the delivery of RGRIP1 cDNA or GUCY2D gene restore the normal subcellular localization of the RPGR and retinal function in mouse model.

Another heterogenous hereditary disorder RP is the most frequent form of inherited retinal dystrophies and caused by the numerous gene mutations associated with this disease, such as RHO gene-encoding rhodopsin, RPE65 gene, RPGR, and the genes encoding for mer receptor tyrosine kinase (MERTK) as well as usherin. The AAV2 vector carrying the MERTK gene and RPE-specific promoter has been applied in several studies to improve the RP patients. The clinical trial of sub-retinal injection of AAV-RPRG gene therapy for RP caused by RPRG mutation has been initiated recently. Some modified vectors have been successfully designed and found to be phototactic, which is effective for gene transfer in RP animal models.

Choroideremia is an X-linked chorioretinal dystrophy characterized by progressive vision loss and caused by a mutation in the CHM gene encoding rab escort protein-1 (REP-1). Currently, choroideremia of the mainly gene therapy focuses on the replacement of mutated CHM gene. In preclinical study, the replacement of the REP-1 gene is evaluated via the subretinally injected AAV-REP-1 in a choroideremia murine model and has been proved to be feasible, safe, and effective. With better understanding of the pathology of CHM, gene replacement therapy has been widely used in clinical research and may be an innovative approaches that contribute to the CHM treatment.

As a frontier biotech service provider, Creative Biolabs provides the best-characterized gene therapy development services for our clients. All of the studies are conducted by experienced technicians and select the most appropriate models and advanced techniques to meet your projects. Please contact us for more detailed information and our team will get back to you as soon as possible.


  1. Moore, N., Morral, N., Ciulla, T. and Bracha, P. (2017). Gene therapy for inherited retinal and optic nerve degenerations. Expert Opinion on Biological Therapy, 18(1), pp.37-49.

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