21 results about "Retinal pigments" patented technology

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.

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

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.

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.

This document relates to AAV vectors (e.g., AAV2 vectors). For example, it provides AAV vectors (e.g., AAV2 vectors) containing AAV capsid polypeptides, such AAV capsid polypeptides, nucleic acid molecules encoding such vectors, nucleic acid molecules encoding such AAV capsid polypeptides, host cells containing and / or expressing such nucleic acid molecules, and methods and materials for preparing or using such vectors and / or AAV capsid polypeptides comprising the amino acid sequences shown in Table 1A (or variants thereof) or Formula A, Table 1B (or variants thereof) or Formula B, or Table 1C (or variants thereof) or Formula C.

Methods of treating ocular diseases (e.g., neovascular age-related macular degeneration, age-related macular degeneration, diabetic macular edema, choroidal neovascularization (including pathologic myopic choroidal neovascularization (mCNV), angiod streaks, choroiditis (including choroiditis secondary to ocular histoplasmosis), idiopathic degenerative myopia, retinal dystrophy, rubeosis iridis, pseudoxanthoma elasticum, and trauma), macular edema following retinal vein occlusion, central retinal vein occlusion, cystoid macular edema, glaucoma (e.g., neovascular glaucoma), polypoidal choroidal vasculopathy, retinopathy of prematurity, ocular von Hippel Lindau disease, diabetic retinopathy, and retinitis pigmentosa) by administering to a human patient an amount and frequency of gomiliximab or a pharmaceutically acceptable salt, solvate, or ester thereof sufficient to treat the ocular disease are provided.

------10 / 06 / 2565------(OCR) This invention provides an improved method for the production of highly purified retinal epithelial (RPE) cells by transforming multievolutionary stem cells. ------------ This invention provides an improved method for the production of highly purified retinal pigment epithelial cells (RPE). By transforming stem cells that are capable of multiple evolutionary processes.

The application discloses a gene panel for detecting retinal pigment degeneration and application thereof, the gene panel comprising PRPF31, TFPT, NDUFA3, OSCAR, USH2A, EYS, RPGR, RHO, RP1, ABCA4, RDH12, CRB1, CNGA1, SNRNP200, CERKL, PDE6B, PROM1, CEP290, RP2, CYP4V2, RPE65, PRPF6 and CNGB1. The gene panel comprises PRPF31 and its upstream and downstream genes, and covers high-frequency mutation genes in an RP population. Through high-density probe design on target regions of the genes, not only the detection cost is reduced, but also precise diagnosis of clinical typing of RP and comprehensive genetic evaluation of PRPF31-RP patients can be realized.

This application relates to a method for inducing stem cells to differentiate into retinal pigment epithelial cells (RPE), comprising the following steps: culturing the pluripotent stem cells in a first culture medium before the pluripotent stem cells exhibit cell state decline after cell culture, wherein the first culture medium comprises DMEM medium, serum, β-mercaptoethanol, non-essential amino acids (NEAA), and L-glutamine. The method described in this application can significantly improve the efficiency of differentiation into RPE.

This invention discloses a method for inducing differentiation and preparing retinal pigment epithelial cells (iPSCs), comprising: treating iPSCs with a first-stage induction differentiation medium, changing the medium daily; discarding the medium, adding a second-stage induction differentiation medium, treating the cells again, changing the medium daily, and picking the cells into matrix gel-coated well plates; discarding the medium, adding a third-stage induction differentiation medium, allowing the cells to adhere, changing the medium again, and allowing the cells to proliferate and grow during this period, while removing contaminating cells. This invention achieves the directed differentiation of iPSCs into retinal pigment epithelial cells through different stages of induction; this method uses a novel culture medium formulation, providing a new approach and method for stem cell differentiation and directed culture.

Among other things, the present disclosure provides compositions, e.g., isolated nucleic acids, vectors, and recombinant adeno-associated viruses (rAAVs), comprising a nucleic acid sequence encoding a PRPF31 polypeptide. In some embodiments, the present disclosure provides methods of increasing expression of PRPF31, e.g., in a subject. In some embodiments, the present disclosure provides methods of treating retinal degeneration in a subject. In some embodiments, the present disclosure provides methods of treating retinitis pigmentosa (RP) in a subject.

The present application belongs to the technical field of biology, and particularly relates to a construction of a retinal pigment degeneration disease model and application thereof. The construction method of the present application adopts a gene editing technology to make the 3rd exon of the CNGA1 gene of a non-human animal model to be mutated, and a retinal pigment degeneration disease model simulating a human c.253delC mutation can be constructed. The retinal pigment degeneration disease model can well simulate the disease caused by the mutation in the human body, and provides a good animal model for mechanism research and drug screening of the disease.

High efficiency methods for producing retinal pigment epithelial cells from induced pluripotent stem cells (iPSCs) are disclosed herein. The iPSCs are produced from somatic cells, including retinal pigment epithelial (RPE) cells, such as fetal RPE stem cells. In some embodiments, the iPSC include a tyrosinase promoter operably linked to a marker. Methods are disclosed for using the RPE cells, such as for treatment. Methods for screening for agents that affect RPE differentiation are also disclosed.

This invention provides a frame-type upconversion infrared visualization glasses, its fabrication method, and its applications, relating to the fields of infrared visualization materials and wearable optical devices. The optical lens of the glasses comprises, from the incident side to the wearer side, an infrared anti-reflection layer, an upconversion core-shell luminescent layer, a visible light gain layer, an infrared reflective layer, and a protective eye layer. This invention uses mesoporous SiO2 nanospheres as the core to construct the upconversion core-shell luminescent layer, and combines it with the infrared anti-reflection layer, visible light gain layer, infrared reflective layer, and protective eye layer to form a five-layer optical enhancement structure. This structure is integrated into the frame lens using a layer-by-layer injection molding and graded curing process, achieving multiple recycling of near-infrared light, efficient loading of rare-earth luminescent components, a significant improvement in upconversion luminescence efficiency, and a synergistic enhancement of visible light output brightness and uniformity on the wearer side. It has broad application prospects in the field of visual assistance for patients with retinitis pigmentosa and late-stage glaucoma.

The present disclosure provides methods of editing RHO using a prime editor (e.g., for correcting a P23H mutation in a RHO protein) and a pegRNA. Such methods may be useful for treating or preventing retinitis pigmentosa. The present disclosure also provides pegRNAs, ngRNAs, complexes, systems, and compositions for editing RHO and treating or preventing retinitis pigmentosa. Polynucleotides, vectors, cells, and kits for editing RHO and treating or preventing retinitis pigmentosa are also provided herein.

Provided herein are methods for the treatment of retinitis pigmentosa in a human that comprises administering to the human a therapeutically effective amount of N-acetylcysteine amide (NACA).

The application discloses a method for establishing a concurrent cataract mouse model, and the mouse model is obtained by retinal-specific knockout of a Phb2 gene. flox / flox The Six3-Cre mouse appears a degenerative retinal disease phenotype at the age of 6-8 weeks, fundus examination shows that the retina is atrophic and thin, the structure of each layer is disordered, the distribution of retinal pigment is disordered, and electrophysiological examination shows that the mouse has abnormal vision function. The mouse appears lens opacity at the age of 8-10 months, and shows a concurrent cataract phenotype. Therefore, the model mouse can better simulate the phenotype of concurrent cataract in clinic, and can be used as an animal model for concurrent cataract related research.

Among other things, the present disclosure provides RHO oligonucleotides, compositions, and methods. In some embodiments, provided oligonucleotides comprise nucleobase modifications, sugar modifications, internucleotidic linkage modifications and / or patterns thereof, and have improved properties, activities and / or selectivities. In some embodiments, the present disclosure provides RHO oligonucleotides, compositions and methods for preventing and / or treating RHO-related conditions, disorders or diseases, such as retinopathy (e.g, retinal degeneration, retinal degenerative disease, retinal degenerative disorder, inherited retinal degenerative disorder, retinitis pigmentosa, autosomal dominant retinitis pigmentosa, etc.).

This invention provides an experimental apparatus and method for testing photodamage to retinal pigment epithelial cells. The apparatus comprises an experimental chamber, an experimental platform, a heating mechanism, a light source mechanism, and a photodamage adjustment mechanism. Multiple cell culture dishes are mounted on the experimental platform. The heating mechanism is located below the cell culture dishes within a bottom heating chamber. The light source mechanism includes a light source control head for generating and adjusting the experimental light source, with the control head facing the experimental platform. The photodamage adjustment mechanism is installed on the experimental platform at a position corresponding to the cell culture dishes, and assists in adjusting the experimental light irradiating the cell culture dishes. This invention utilizes the combined operation of the light source mechanism and the photodamage adjustment mechanism to achieve continuous adjustment of the light spot diameter, flexibly simulating photodamage lesions of different scales from microfocal to diffuse, meeting the needs of various disease research.