| Summary | Study OpCT-001-101 is a Phase 1/2a first-in-human, multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor (PR) disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups. | 
                                
                    | Which differentiated cell type is used | 
            
                                                                
            | Label | photoreceptor cell |  
            | Link | http://purl.obolibrary.org/obo/CL_0000210 |  
            | Description | A cell specialized in detecting light stimuli that are involved in visual perception.; Photoreceptor cells are specialized types of neurons found in the retina, responsible for the vital task of converting light into signals that can stimulate a visual response. These cells are called photoreceptor cells because they are light-sensitive, enabling vision in conditions ranging from extremely dim to very bright light. There are two main types of photoreceptor cells in the human eye: rods, which are highly sensitive to light and allow for vision in low-light conditions, and cones, which are responsible for color vision and function best in bright light.
The photoreceptor cells function by absorbing light rays and converting them into electrical signals. These signals are then sent to the brain via the optic nerve and are interpreted as visual images. This process begins when the photopigment (a light-sensitive compound) found in photoreceptor cells absorbs light, which triggers a chemical reaction. This reaction leads to changes in the photopigment's configuration and, consequently, alters its electrical properties. This change initiates a signal that is transmitted through the cell and onwards to connecting neurons.
Furthermore, photoreceptor cells exhibit a unique and highly specialized structure that reflects their function. Rods, named because of their elongated, rod-shaped structure, consist of an outer segment filled with disks containing rhodopsin, the photopigment responsible for detecting light. Cones, on the other hand, are more conical and have three different types of photopigments, each one sensitive to a different range of wavelengths, allowing for the perception of color. Given the importance of photoreceptor cells in vision, damage or degeneration to these cells can lead to vision impairment or blindness, highlighting their key role in visual perception.
(This extended description was generated by ChatGPT and reviewed by the CellGuide team, who added references, and by the CL editors, who approved it for inclusion in CL. It may contain information that applies only to some subtypes and species, and so should not be considered definitional.) |  |