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The retinal pigment epithelial cells (RPE) make up a polarized monolayer in the vertebrate eye that separates the neural retina from the choroid, and performs a crucial role in retinal physiology by forming a blood-retinal barrier and closely interacting with photoreceptors to maintain visual function.  Many ophthalmic diseases, such as age-related macular degeneration, are associated with a degeneration or deterioration of the RPE. 

Interleukin (IL)-34 is a homodimer that is produced mainly by keratinocytes, neuronal cells and regulatory T cells (Tregs). It is believed to play important roles in chronic inflammation and the homeostasis of microglia. Currently, there is no effective treatment for many types of retinal degeneration. An improved treatment of autoimmune uveitis is also needed, as current uveitis treatment primarily uses steroidal anti-inflammation medication, which may produce significant unwanted side effects in long-term use.

Regulatory B-cells (Breg) play an important role in reducing autoimmunity and reduced levels of these cells are implicated in etiology of several auto-inflammatory diseases. Despite their impact in many diseases, their physiological inducers are unknown.  Given that Bregs are a very rare B-cell, identifying factors that promote their development would allow in vivo modulation of Breg levels and ex-vivo production of large amounts of antigen-specific Bregs to use in immunotherapy for auto-inflammatory diseases.
 

Proinflammatory T-helper 17 cells (Th17) play important roles in host immune defense against infection, but uncontrolled activation of these cells, known as the Th17 response, may cause autoimmune and autoinflammatory diseases (uveitis, multiple sclerosis, rheumatoid arthritis, and Crohn’s disease) through the effects of Th17 lineage cytokines (such as, IL-17F, IL-22 and GM-CSF). Importantly, IL-17A (a proinflammatory cytokine) represses other Th17 lineage cytokines by upregulating the regulatory cytokine IL-24.

The limited choice in cell types available for in vitro studies has become an obstacle in hibernation research. 

Researchers at the National Eye Institute for the first time have successfully established iPSC line(s) from a mammalian hibernator, which can be potentially used to generate various cell types and tissue models for in-depth mechanistic studies of hibernation and coldness tolerance in vitro. 

Researchers at the National Eye Institute (NEI) have discovered an invention describing a composition and method(s) of using such composition for preserving viability of cells, tissues, or organs at a low temperature (around 4ºC). Current cold storage solutions or methods for cells, tissues, and organs are suboptimal due to irreversible damage to cold-sensitive tissue or organ transplants that need a longer term of storage for facilitating clinical practices.

Problem: Cryopreservation is a process where living biological materials like cells, tissues, and cell therapies (which are susceptible to damage caused by unregulated chemical kinetics) are preserved by cooling to very low temperatures in the presence of specific cryopreservation media that protects the biological material from damage. In order to be used, the biological material ideally should be thawed in a controlled manner that minimizes damage and desirably brings the material back to a viable state.

The accurate placement of transplanted tissue at a precise position in the retina is difficult but critical for a successful implementation of an ocular surgical intervention. 

Medical clamps currently available are not efficient nor are they sufficiently precise in closure and alignment of the edges of an incision or wound. Many available designs are difficult to use and handle, especially in situations where repeated opening and closure of an incision or wound is required. The functional short-comings of existing clamp designs may result in surgical complications, such as excess loss of fluids and pressure and hemostasis during some procedures.

Summary:  

The National Eye Institute (NEI) seeks research co-development partners and/or licensees for the development of an AAV2-based delivery system or an eyedrop formulation to deliver a Pigment Epithelium-Derived Factor (PEDF) peptide as a gene-agnostic approach to treating inherited retinal diseases.