69 results about "Retinal degeneration" patented technology

This invention relates to methods for improved cell-based therapies for retinal degeneration and for differentiating human embryonic stem cells and human embryo-derived into retinal pigment epithelium (RPE) cells and other retinal progenitor cells.

The present invention relates to novel analogs of choline and methods of use or treatment of neurodegenerative disorders and / or conditions such as Parkinson's disease, Huntington disease, Alzheimer's disease and related disorders such as amyotrophic lateral sclerosis, spinal muscular atrophy, Friedrich's ataxia, Pick's disease, Bassen-Kornzweig syndrome, Refsom's disease, retinal degeneration, Cruetzfelt-Jacob syndrome or prion disease (mad cow disease), dementia with Lewy bodies, schizophrenia, paraneoplastic cerebellar degeneration and neurodegenerative conditions caused by stroke. The present compounds are effective to treat any neurological condition where acetylcholine transmission neurons and their target cells are affected. Compounds according to the present invention are effective to alleviate and / or reverse the effects of a neurodegenerative condition, prevent further deterioration and / or enhance cognition and memory in patients suffering from neurodegenerative disorders, especially Alzheimer's disease.

This invention relates to methods for improved cell-based therapies for retinal degeneration and for differentiating human embryonic stem cells and human embryo-derived into retinal pigment epithelium (RPE) cells and other retinal progenitor cells.

The present invention relates to an improved method of providing photoreceptor function to a cell, for example for use in the treatment of retinal degeneration. The present invention also relates to compositions and kits, in particular for use in such methods.

This invention relates to methods for improved cell-based therapies for retinal degeneration and for differentiating human embryonic stem cells and human embryo-derived into retinal pigment epithelium (RPE) cells and other retinal progenitor cells.

Disclosed is a method for inducing stem cells to differentiate into retinal cells at high yield within a short period of time, without gene implantation and co-culture with retinal tissues, by implementing a differentiation process similar to the in vivo embryonic development in chemically defined conditions. Also, retinal cells including the photoreceptor cells and their progenitor cells, and various types of other retinal cells, generated according to the method, are disclosed. A composition comprising the retinal cells and a method are provided for treating retinal degeneration-related diseases. The differentiated photoreceptor cells, when transplanted into degenerated or injured retinas, can be engrafted and fused within the retinas to prevent or cure retinal degeneration.

A method of treating retinal diseases is disclosed that includes the step of administering an effective amount of a composition including an ASMase inhibitor to a retinal disease patient, wherein at least one disease symptom is either lessened or progression of the symptom is delayed.

Disclosed herein are methods of treating disorders of the retina (e.g., macular degeneration, retinitis pigmentosa, etc.) comprising administering to a subject in need of such treatment a therapeutically effective amount of a compound (for example, an antipsychotic drug) that blocks or diminishes agonist-mediated responses upon binding to either dopamine D2-like receptors or serotonin 5-HT2 receptors.

Intraocular injection of adeno-associated viral (AAV) vectors has been an evident route for delivering gene drugs into the retina. Currently, the vectors need to be injected into the subretinal spacein order to provide gene delivery to cones. In this approach, gene delivery is limited to cells that contact the local "bleb" of injected fluid. Furthermore, retinal detachment that occurs during subretinal injections is a concern in eyes with retinal degeneration. Here, the inventors establish several new vector-promoter combinations to overcome the limitations associated with AAV-mediated cone transduction in the fovea with supporting studies in mouse models, human induced pluripotent stem cell-derived organoids, post-mortem human retinal explants and living macaques. They show that an AAV9variant provides efficient foveal cone transduction when injected into the subretinal space several millimeters away from the fovea, without detaching this delicate region. The delivery modality relies on a cone-specific promoter and result in high-level transgene expression compatible with optogenetic vision restoration. Accordingly, the present invention relates to method of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector comprising a VP1 capsid protein asset forth in SEQ ID NO: 11 and the polynucleotide of interest under the control of the pR1.7 promoter as set forth in SEQ ID NO: 12.

Tissue replacement implants are disclosed that include polarized retinal pigment epithelial cells on a poly(lactic-co-glycolic acid) (PLGA) scaffold, wherein the PLGA scaffold is 20-30 microns in thickness, has a DL-lactide / glycotide ratio of about 1:1, an average pore size of less than about 1 micron, and a fiber diameter of about 150 to about 650 nm. Also disclosed are methods of treating a subject with a retinal degenerative disease, retinal or retinal pigment epithelium dysfunction, retinal degradation, retinal damage, or loss of retinal pigment epithelium. These methods include locally administering to the eye of the subject the tissue replacement implant. In further embodiments, methods are disclosed for producing the tissue replacement implant.

The invention relates to an NOX2 gene defect rd1 mouse model as well as a preparation method and application thereof, and belongs to the technical field of biology. The invention provides a method for preparing an NOX2 gene defect rd1 mouse model, which comprises the following steps: hybridizing and screening an NOX2 gene knockout mouse and an rd1 mouse to obtain the NOX2 gene defect rd1 mouse model. The loss of retinal outer nuclear layer photoreceptor cells of the NOX2 gene defect rd1 mouse model prepared by using the method is obviously delayed, the activation of microglia cells is obviously inhibited, and the expression of NOX2 in the microglia cells is obviously reduced, so that the NOX2 activation in the microglia cells is further verified to be a pathogenic mechanism of hereditary retinal degeneration; in addition, the invention provides a basis for screening and researching and developing medicines for treating and / or preventing retinal degeneration, which are clearer in action mechanism, stronger in specificity and better in market and clinical application prospect, and has a great application prospect.

The invention discloses the application of hsa-miR-382-5p in preparing a kit for diagnosing retinal degenerative diseases. A diagnostic kit for retinal degenerative diseases, comprising a reagent for detecting the expression level of hsa-miR-382-5p. Application of a substance inhibiting the expression of hsa-miR-382-5p in the preparation of a drug for treating retinal degenerative diseases. The study found that the increased expression of hsa-miR-382-5p can be used as an auxiliary diagnosis basis for retinal degenerative diseases and help realize the early diagnosis of retinal degenerative diseases. The damaging effect of hsa-miR-382-5p on the retina suggests that it can be used to prepare and screen drugs for treating retinal degenerative diseases. The invention provides a new detection marker and therapeutic target for the retinal degenerative disease, and provides brand new molecular information and biological basis for the diagnosis and treatment of the disease.