The field of genetics has made great progress since the concept in the 1970s. With the identification of a variety of opsins from single-cell organisms, the emergence of safe and effective viral vectors for transmitting genetic materials and the development of many transgenic animal models, optogenetics has become more and more close to clinical application. The field of optogenetics has been expanding rapidly. Genetic methods combined with optical technology are used to achieve the acquisition or loss of neuronal circuit function, and strive to restore the visual function of blinding diseases such as retinitis pigmentosa (RP). Independent progress of opsin engineering, virus packaging; targeting and expression. Finally the molecular control of remodeling is the key to the final clinical application of optogenetic therapy for degenerative retinal diseases.
Creative BioMart provides various solutions for clients who desire to do retinal disorders therapeutic research. We have the ability to provide the construction services of cell lines, expression systems and model organisms.
Viral vector design and delivery
Most of the work in this field includes a group of photosensitive retinol binding proteins called opsin. Opsin couples photon absorption to molecular signal chains that control the ionic current of cells. Targeting opsin gene to surviving retinal cells is the basis of successful optogenetic therapy. Viral delivery, mainly adeno-associated virus, using intravitreal injection for internal retinal cells and subretinal injection for external retinal cells, has proved successful in many models.
Related models of photogenetic therapy
Open luminescence genetic therapy requires preclinical testing in relevant in vivo and in vitro models of hereditary retinal diseases. When studying photogenetic therapy, the selection of models is essential to understand its transformation potential and may affect the degree of therapeutic effects observed. Therefore, the efficacy of a specific optogenetic sensor may depend on the expression level achieved in the target cells, which in turn depends on the research model used.
Common mouse models of hereditary retinal degeneration
The mouse model rd1 with natural mutation of photoreceptor specific gene pde6b has good characteristics and is the most commonly used model for preclinical testing of photogenetic therapy.
Human-derived retinal organoids
The use of model organisms has many uses. In vivo testing has many benefits, especially in the field of optogenetics, which can realize the evaluation of visual function and processing. However, it is important to consider the benefits of other models in preclinical testing. In recent years, protocols for generating retinal organoids from human iPSCs have been developed, which enables the formation of culture structures derived from human cells that reflect the cell types and tissues of the neuroretina. These retinal organoids not only provide a very useful tool for preclinical testing of optogenetic therapy, but also are widely used in the field of retinal gene therapy.
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