155 results about "Retinal pigments" patented technology

Described herein are certain methods of treating a subject with retinitis pigmentosa, the method comprising administering to the subject a therapeutically effective dose of a rhodopsin corrector molecule thereby treating the retinitis pigmentosa.

The present invention relates to an exogenous target gene expression cassette for delivering an exogenous target gene to the retina, in particular AIPL1 to retinal pigment epithelial cells and photoreceptor cells, comprising an IRBP enhancer sequence, a rhodopsin kinase (RK) promoter sequence and a CAG intron sequence, which are operatively linked, and an exogenous target gene. The present invention also relates to a recombinant adeno-associated viral vector comprising a viral capsid comprising a capsid protein or a capsid protein variant and a viral vector genome comprising an expression cassette encoding for specifically expressing an exogenous target gene in retinal pigment epithelial cells and photoreceptor cells. The recombinant adeno-associated virus vector can be used for relieving or treating retinal degenerative eye diseases by intravitreal administration or subretinal administration.

Provided herein are compositions and methods for treating patients with Usher syndrome associated retinitis pigmentosa (UARP) comprising identifying that the subject has UARP and administering to the animal or human an effective amount of an N-acetylcysteine amide (NACA) sufficient to protect vision and inhibit degradation of Ellipsoid Zone (EZ) area and retinal sensitivity. In one example, the NACA formulation is doses at 50, 100, 200, 201, 210, 225, 250, 275, 300, 350, 400, 450, 500, 600, 700, 750, 800, 900, or 1,000 mg / day.

The invention provides universally acceptable “off-the-shelf” hypoimmunogenic pluripotent cells and differentiated cardiac, endothelial, neuronal, islet, or retinal pigment cells thereof. Such hypoimmune cells are used to treat patients in need thereof. The cells lack major immune antigens that trigger immune responses and are engineered to avoid phagocytic endocytosis.

The invention belongs to the technical field of nano-drugs, and discloses a multifunctional nano-drug delivery system for treating neovascular eye diseases and a preparation method of the multifunctional nano-drug delivery system. The nano drug delivery system disclosed by the invention is a composite nano preparation which takes dendritic mesoporous silica nanoparticles as a carrier and co-loads cerium zirconium oxide nano enzyme and an anti-VEGF (vascular endothelial growth factor) drug, various active oxygen can be efficiently removed, oxygen is continuously released through catalytic decomposition of hydrogen peroxide, a retina hypoxia microenvironment is improved, and the retina hypoxia effect is improved. According to the present invention, the anti-VEGF drug delivery system can reduce the oxidative stress induced retinal pigment epithelial cell apoptosis rate, reduce the inflammatory reaction, and simultaneously achieve the slow release and the controlled release of the anti-VEGF drug, and the drug effect maintenance time can achieve more than 60 days, such that the multi-target synergistic treatment can be achieved through the synergistic regulation of the multiple pathological links such as oxygen deficit-oxidative stress-inflammation-angiogenesis, and the anti-VEGF drug delivery effect can be achieved. The choroidal neovascularization can be effectively inhibited by single intravitreal injection, and a new strategy of multi-target collaborative treatment is provided for neovascular macular degeneration and other eye diseases.

The invention provides a method for inducing human pluripotent stem cells to be differentiated into retinal pigment epithelial cells and application. The method comprises the following steps: performing adherent culture on pluripotent stem cells to serve as differentiation starting cells; the differentiation starting cells are cultured in stages with different culture media to obtain the retinal pigment epithelial cells, and the culture media in at least one stage comprise a WNT non-classical pathway activator. The invention further provides a composition and a culture medium for inducing the human pluripotent stem cells to be differentiated into the retinal pigment epithelial cells, the differentiation efficiency of the RPE can be remarkably improved, the cell harvesting purity is improved, and the composition and the culture medium are suitable for large-scale industrial production.

The invention discloses a construction method and application of a Prpf6 gene mutation induced single dose insufficient RP mouse model, and belongs to the technical field of molecular biology and biomedicine. The mouse model constructed by using the method provides a convenient, reliable and economic means for researching the relationship between PRPF6 gene mutation and retinitis pigmentosa diseases and the pathogenesis of the PRPF6 gene mutation, and provides an effective research model capable of stably inheriting in the research on the aspects of RP pathogenesis, treatment methods, drug screening and the like. The animal model is subjected to transcriptome and a series of phenotype detection, wherein a transcriptome result shows that Prpf6 gene knockout mouse retina has occurred a certain degree of metabolic abnormality, electrophysiological detection, fundus examination and Hamp; e staining results show that the shapes and functions of the retinal cell layer and the retina of the mouse are damaged, and the disease phenotype of RP is met.

The present invention provides a pharmaceutical composition for preventing or treating ophthalmic diseases, comprising a nuclear receptor subfamily 2 group F member 1 (NR2F1) inhibitor. The NR2F1 inhibitor of the present invention rejuvenates retinal cells by inhibiting the expression of the NR2F1 gene or the activity of the NR2F1 protein, and thus has the effects of reducing cellular senescence of retinal pigment epithelial cells and restoring visual function.

To provide a pharmaceutical composition for restoring lysosomal function of retinal pigment epithelial cells.SOLUTION: Provided is a pharmaceutical composition for restoring lysosomal function of retinal pigment epithelial (RPE) cells in a subject, comprising a polypeptide comprising βA1-crystallin or a nucleic acid encoding the polypeptide.SELECTED DRAWING: Figure 1

The invention discloses a xanthophyll eye-protecting composition and application thereof, and belongs to the field of traditional Chinese medicine technology and food. The eye protection composition is prepared from the following raw materials in parts by weight: 1 to 50 parts of lutein or lutein ester, 1 to 10 parts of zeaxanthin, 1 to 650 parts of anthocyanin, 1 to 5 parts of docosahexaenoic acid, 1 to 40 parts of radix puerariae, 1 to 40 parts of fructus lycii peptide, 1 to 10 parts of vitamin C and 1 to 15 parts of flos inulae nervosae. According to the composition, the raw materials with the eye protection effect are compounded with the specific new food raw materials including the inula nervosa, the bead peptide powder and the chimonanthus salicifolius, blue light damage of human retinal pigment epithelial cells ARPE-19 and inflammatory response caused by the blue light damage can be effectively relieved, and the composition can have the better eye protection effect.

The invention discloses a double-drug-loading cationic mixed micelle as well as a preparation method and application thereof, and belongs to the field of pharmaceutics. The micelle is composed of a cationic polymer PEI-stearamide (PSA), an amphiphilic polymer DSPE-PEG2000, a hydrophobic drug resveratrol (RES) and a hydrophilic drug catalase (CAT). The preparation method comprises the following steps: 1) synthesizing PSA; the preparation method comprises 1, dissolving RES, PSA and DSPE-PEG2000 in an organic phase, injecting the obtained solution into a water phase, and carrying out rotary evaporation to obtain RES-loaded micelle (RES / PSA-PEG), and 3, adding a CAT solution to the micelle solution in a dropwise manner to obtain co-loaded micelle (RES / PSA-PEG / CAT). The micelle can effectively penetrate a corneal barrier and target a retinal pigment epithelial layer and a choroidal membrane.Through eye drop administration, RES anti-oxidation and anti-inflammatory effects and CAT hydrogen peroxide removal effects can be synergistically exerted, and an AMD curative effect can be substantially enhanced.

The invention discloses application of cnidium lactone in preparation of a medicine for treating AMD or corneal injury, and belongs to the field of biological medicine. It is found for the first time that the cnidium lactone can inhibit sodium iodate induced retinal pigment epithelial cell injury, and it is also found for the first time that the cnidium lactone can promote injured cornea repair; based on the discovery, the invention respectively provides application of cnidium lactone in preparation of medicines for treating age-related macular degeneration and preparation of medicines for treating corneal injury. Experimental results show that cnidium lactone can remarkably reverse the retinal pigment epithelial cell degradation trend caused by age-related macular degeneration and can also remarkably promote corneal notch healing. The invention provides a new optional medicine for treating age-related macular degeneration and corneal injury, expands the application range of cnidium lactone, and has outstanding clinical practical value and wide application prospect.

The invention belongs to the field of RPE cell preparations, and relates to a retinal pigment epithelial cell spherical preparation which is obtained by the following steps: step 1, obtaining RPE cells; and 2, inoculating the RPE cells into a cell culture plate by using an inoculation culture medium, and culturing after centrifugal precipitation, so that a plurality of RPE cells are aggregated to form RPE cell balls with uniform sizes. The cell preparation form obtained by the scheme not only can avoid injection leakage of the subretinal space, but also has the advantages of being convenient to inject and uniform in cell expansion, so that preclinical needs are met, and an important basis is provided for preparation of a clinical-grade RPE cell preparation and subsequent further development of clinical research.

The application relates to the technical field of ophthalmic surgical instruments, in particular to an ophthalmic internal limiting membrane tampon, which can protectively and automatically rebound when the tampon is operated with excessive force during a'mechanical' tampon process, so as to prevent iatrogenic retinal pigment epithelial layer damage at the bottom of a macular hole. The device comprises a sleeve-shaped handle, a spring and a probe are arranged in the handle along the length direction of the handle; the probe comprises a probe main body and a probe base, and the two ends of the probe base are connected with the spring and the probe main body respectively; an outer thread is arranged on the outer side of the probe base, and a tensioning wheel is arranged on the handle; the outer thread is divided into at least three thread regions arranged along the length direction of the probe base, including a first thread region, a second thread region and a third thread region; the threads in the first thread region, the second thread region and the third thread region correspond to first threads, second threads and third threads respectively; and the diameters of the second threads are all larger than the diameters of the first threads and the third threads.

The present invention relates to: an AAV-based PDE6B viral vector for treating retinitis pigmentosa, the AAV-based PDE6B viral vector containing a tissue-specifically expressed PDE6A promoter; and a use of thereof, and provides a gene therapy for treating retinitis pigmentosa caused by PDE6B gene deficiency. The in vivo therapeutic efficacy of seven types of AAV5-PDE6B vectors was confirmed using an AAV by using a PDE6A promoter that is tissue-specifically expressed in photoreceptor rod cells that develop retinitis pigmentosa. An AAV5-PDE6A-450-PDE6B vector was selected as a candidate due to exhibiting strong tissue-specific expression in photoreceptor rod cells under even off-target conditions, unlike the gene expression characteristics of an AAV5-CMV-PDE6B vector, and was tested so as to be usable in the development of a gene therapeutic agent for treating PDE6B-deficient retinitis pigmentosa patients. Therefore, the present invention, related to AAV5-PDE6B for retinitis pigmentosa treatment and containing a tissue-specifically expressed PDE6A promoter, provides retinitis pigmentosa patients with an important treatment option having improved safety, and can be expected to have fundamental therapeutic effects compared to conventional treatments.

The present application relates to a kind of microglial cell P2Y6R target point inhibitor and its application, belong to biological medicine technical field.For the problems such as lack of effective treatment means for retinal pigment degeneration, abnormal activation of microglial cell accelerates course and lack of specific intervention target point, the application of inhibitor with P2Y6R as target point in preparation treatment retinal pigment degeneration drug is proposed, especially siRNA and shRNA for knocking down microglial cell P2ry6 gene are provided, and AAV vector containing microglial cell specific promoter is constructed, the specific silencing of P2ry6 in retina is realized.The present application can significantly inhibit abnormal activation of microglial cell and inflammatory response, reduce photoreceptor apoptosis, improve retina structure and visual function, and provide a new strategy for targeted treatment of retinal pigment degeneration.

To provide compositions containing RPE cells or RPE monolayers, as well as methods and materials for making RPE cells or RPE monolayers from, for example, stem cells (e.g., iPSCs).SOLUTION: Disclosed is a method for making a retinal pigment epithelium monolayer, the method comprising, or consisting essentially of, culturing stem cells in a container having a surface coated with fibrinogen, wherein the surface is coated with greater than 3 μg / mL of fibrinogen, wherein the cells are in contact with the fibrinogen, and wherein the cells form the retinal pigment epithelium monolayer.SELECTED DRAWING: None

The application discloses a method for constructing a RP mouse model caused by Prpf6 gene mutation and haploinsufficiency and application thereof, and belongs to the field of molecular biology and biomedical technology. The mouse model constructed by the method provides a convenient, reliable and economical means for studying the relationship between the PRPF6 gene mutation and the retinal pigment degeneration disease and the pathogenic mechanism thereof, and provides a stable hereditary effective research model in the research of the pathogenic mechanism, treatment method and drug screening of RP. The animal model is subjected to transcriptome and a series of type detection, wherein the transcriptome result shows that the retina of the Prpf6 gene knockout mouse has a certain degree of metabolic abnormality, and the results of the electro-physiological detection, fundus examination and H&E staining show that the cell layer of the mouse retina and the shape and function of the retina are damaged, which is consistent with the disease phenotype of RP.

Disclosed herein are mesoporous ceria nanoparticles for the prevention, treatment, or alleviation of ophthalmic diseases. In one aspect, having mesoporous structure with high surface area and pore size and mimicking the catalytic properties of catalase and superoxide dismutase (SOD), the mesoporous ceria nanoparticles of the present invention have excellent ability to scavenge reactive oxygen species, exhibit anti-inflammatory effects, demonstrate biocompatibility without causing acute side effects or ocular toxicity in the retina, and are recognized to provide protective effects against cellular damage and protect damaged retinal pigment epithelial (RPE) cells and photoreceptors under high oxidative stress. Therefore, the nanoparticles can be utilized for the prevention, treatment, or alleviation of macular degeneration and various oxidative stress-mediated ophthalmic diseases.

An isolated or purified antisense oligomer for modifying pre-mRNA splicing in the CNOT3 gene transcript or part thereof.

The invention discloses application of a DKK1 inhibitor in preparation of a medicine for treating dry age-related macular degeneration. According to the application disclosed by the invention, the expression of DKK1 in dry AMD cells and animal models induced by sodium iodate (NaIO3) is obviously improved. The form of the retinal pigment epithelial cells can be protected and the death rate can be reduced by using the siRNA of the targeted DKK1 in an in-vitro experiment, and the plaque loss of the retinal pigment epithelial cells can be relieved and the retinal pigment epithelial cells can be protected by using the shRNA of the targeted DKK1 in an in-vivo experiment. Therefore, the invention provides a new therapeutic target and therapeutic strategy for dry AMD, and has important clinical application value.

The present invention addresses the problem of providing a protective agent for retinal pigment epithelial cells, which is useful for the prevention or treatment of age-related macular degeneration. A protective agent for retinal pigment epithelial cells according to the present invention, which is a solution for the problem, contains, as an active ingredient, a peptide in which a protein transduction domain is attached to the N-terminus of a peptide having an inhibitory effect on mitochondrial calpains. A specific example of the peptide having an inhibitory effect on mitochondrial calpains is a peptide comprising the amino acid sequence (PDALKSRTJR) represented by SEQ ID NO: 1 (wherein J represents L or I). A specific example of the protein transduction domain is a peptide comprising an amino acid sequence that is at least a part of the amino acid sequence for the transduction domain of HIV-1 Tat.

Provided are a production method of retinal pigment epithelial (RPE) cells that improves differentiation induction efficiency of pluripotent stem cells into RPE cells, and can provide highly pure RPE cells by a simple and easy operation in a short period, a culture method of RPE cells that can stably grow and culture a cell, a toxicity / efficacy evaluation method using RPE cells useful for transplantation therapy, and a therapeutic drug for a retinal disease. The invention relates to a production method of RPE cells, comprising adhesion culture of human pluripotent stem cells using a culture substrate coated with a laminin-E8 fragment, a culture method of RPE cells, comprising adhesion culture of RPE cells using a culture substrate coated with a laminin-E8 fragment, a toxicity or efficacy evaluation method using RPE cells obtained by producing or culturing by the method, and a therapeutic drug for a retinal disease, containing the RPE cells.

The present disclosure provides an augmented reality (AR) device for patients with retinitis pigmentosa and macular degeneration comprising a map providing unit, a region setting unit, and a region display unit, wherein the map providing unit sequentially drives a plurality of scanning points included in a display of wearable glasses to provide a scotoma map wherein a user's scotoma is checked according to the user's input signal, the region setting unit sets the user's scotoma region based on the scotoma map; the region display unit displays an external image corresponding to the scotoma region in a display region included in the display.

The present invention provides a method for preparing retinal pigment epithelial cells, comprising (1) a first step of subjecting pluripotent stem cells to suspension culture in a serum-free medium to form aggregates of the pluripotent stem cells, (2) a second step of subjecting the aggregates of the pluripotent stem cells to suspension culture in a serum-free medium to form aggregates of the pluripotent stem cells. (1) a third step in which the aggregate formed in step (1) is subjected to suspension culture in a serum-free medium or a serum-containing medium, each of which does not contain a substance acting on the Sonic hedgehog signal transduction pathway and contains a substance acting on the BMP signal transduction pathway, to obtain an aggregate containing retinal progenitor cells; the aggregate formed in step (2) is subjected to suspension culture in a serum-free medium or a serum-containing medium that does not contain a substance that acts on the Sonic hedgehog signal transduction pathway and a substance that acts on the BMP signal transduction pathway, but contains a substance that acts on the Wnt signal pathway, thereby obtaining an aggregate containing retinal pigment epithelium cells.

Disclosed is an agent for the treatment of a common form of autosomal dominant retinitis pigmentosa (ADRP) wherein the agent comprises a first nucleotide sequence encoding a CRISPR-related (Cas) endonuclease bound to an NG or NNGRRT PAM (Primal Spacer Adjacent Motif) sequence, and a second nucleotide sequence encoding or comprising a guide RNA (gRNA) capable of forming a CRISPR-Cas complex with the Cas endonuclease, wherein the gRNA specifically targets a target mutant allele selected from the group consisting of RHOP23H and NR2E3G56R.

Provided herein are AAV2 vectors encoding SEQ ID NO:4 as optogenetic proteins and methods of use, for example, for retinitis pigmentosa.

The invention discloses a novel application of Selpatinib, and belongs to the field of biological medicines. It is found for the first time that the Selpatinib inhibits retinal pigment epithelial cells from being converted into mesenchymal cells, and the inhibition action mechanism of the Selpatinib is further explored as follows: the TGF beta signal transduction process is stopped by inhibiting the combination of RET and a TGFBR1 receptor I and then blocking RET from entering a nucleus (membrane), and the action mechanism of the Selpatinib RET inhibitor is broken through. The invention discovers the new application of the Selpatinib in treating the ophthalmic diseases related to epithelial-mesenchymal transition of retinal pigment epithelial cells, not only broadens the medicinal application of the Selpatinib and improves the medicinal value of the Selpatinib, but also provides a more targeted new medicine for the ophthalmic diseases related to EMT (epithelial-Mental Transformation), so that the application of the Selpatinib in treating the ophthalmic diseases related to the EMT is developed. The research and development of medicines for inhibiting epithelial-mesenchymal transition related diseases are promoted, and the application prospect is relatively great.

This invention discloses the application of the VDAC2 gene or VDAC2 protein as a target in screening drugs for the treatment of retinitis pigmentosa, relating to the field of biomedical technology. The drug is one that reduces the expression level of the VDAC2 gene or inhibits the expression level or activity of the VDAC2 protein; the CDS sequence of the VDAC2 gene is shown in SEQ ID NO.1. This invention uses a rod cell-specific promoter to inhibit VDAC2 expression, which can significantly improve the survival rate of local photoreceptor cells. This method intervenes in the early stages of the disease, conforming to the natural course of RP. It strengthens the function of rod cells before a large number of photoreceptor cells die, delaying their degenerative process and indirectly protecting cone cells, thereby prolonging the patient's effective visual retention time. This overcomes the shortcomings of existing gene therapies, which are mainly applicable to late-stage or specific mutation types, and provides a therapeutic basis for retinitis pigmentosa.

The invention belongs to the technical field of medicines, and particularly relates to application of vitamin B12 in preparation of a medicine for treating retinitis pigmentosa. Specifically, based on the high conservative property of human PRPF31 and caenorhabditis elegans prp-31 genes, a prp-31 gene knock-down and mutation nematode disease model is constructed, and the model can reproduce multiple pathological phenotypes such as oxidative stress, lysosome dysfunction and apoptosis caused by PRPF31 defects. Experiments prove that the active form (such as mecobalamin) of the vitamin B12 can actively pass through the blood retina barrier by virtue of an endogenous transport mechanism through oral administration, the pathological damage can be reversed by virtue of multi-channel synergy, and the pathological phenotype of a model organism can be remarkably improved, so that the vitamin B12 has a good practical application value.