12 results about "Retinal degeneration" patented technology

This invention discloses a method for establishing a mouse model of outer retinal tubular structures, which is obtained by specifically knocking out the Lss gene in the mouse retina. This invention utilizes CRISPR / Cas9 gene editing technology to construct a mouse model of outer retinal tubular structures by specifically knocking out the Lss gene in the retina. The mouse model of outer retinal tubular structures constructed by this method is similar to the clinical phenotype of this type of retinal disease, providing a relatively ideal experimental animal model for studying the developmental regulation of the mammalian retina and the pathogenesis of retinal degeneration.

Microparticulate (MP) formulations formed from one or more poly(hydroxyacid) polymers having a molecular weight ranging from 5kD to 60kD, and one or more active agents are injected into the eye of a subject to address eye disorders. The MP formulations assure high drug loading and extended delivery of the active agent of six to twelve months. The active agents may include peroxisome proliferator-activated receptor alpha (PPARα) signaling agonists such as PPARα agonist A190 (IUPAC name 3-((4-((4-fluorobenzyl)oxy)- 3- methylbenzyl)amino)benzoic acid). The formulations have therapeutic and protective effects against retinal degeneration diseases such as age-related macular degeneration (AMD), but may also be used for treating or relieving the symptoms of retinal inflammation, retinal neovascularization, retinal vascular leakage, retinopathy of prematurity (ROP), diabetic retinopathy (DR), and diabetic macular edema (DME).

The application discloses a traditional Chinese medicine composition for treating senile macular retinal degeneration and a preparation method thereof. The traditional Chinese medicine composition is prepared from wolfberry fruit, chrysanthemum, milkvetch seed, dendrobium, panax notoginseng, raw safflower, and radix puerariae according to certain weight ratios. The traditional Chinese medicine composition is prepared into an oral preparation and has the effect of treating senile macular retinal degeneration.

Methods for treatment of damaged corneal endothelium are provided, applicable for treatment or protection of corneal endothelium in various circumstances such as oxidative stress, age-related decline in endothelial cell density, inherited or non-inherited degenerative disease such as Fuchs endothelial corneal dystrophy, surgical trauma, increased intraocular pressure (IOP) or contact lens overuse. Further provided are methods for treatment of retinal degeneration such as in diabetic retinopathy, age-related macular degeneration and glaucoma. The methods comprise the transplantation of viable isolated, exogenous, mitochondria systemically and / or directly to the cornea or the retina.

This invention relates to the application of human amniotic epithelial stem cells (hAESCs) in retinal regeneration and repair, belonging to the field of biomedical technology. It aims to address the technical problems of off-target effects, low efficiency, and safety risks associated with existing methods that directly reprogram Müller glial cells (MGs) via viruses. This invention transplants hAESCs into the degenerated subretinal space. hAESCs, through secreting active substances such as matrix metalloproteinases (MMPs), degrade excessively deposited extracellular matrix, improve the pathological microenvironment inhibiting regeneration, and thus promote the reprogramming of endogenous MGs into functional retinal neurons, thereby achieving retinal structural repair and visual function restoration. This invention utilizes highly safe hAESCs and, by remodeling the microenvironment, significantly improves the efficiency and specificity of MG differentiation into functional neurons, providing a new strategy for the treatment of retinal degenerative diseases.

The instant invention provides methods and compositions related to discovery of Free Fatty Acid Receptor 1 (FFA1) as a therapeutic target for treatment or prevention of diseases or disorders of neurons that are characterized by angiogenesis, or of vascular diseases of the eye, retinal degeneration and / or tumors more generally. Therapeutic and / or prophylactic uses and compositions of known FFA1 inhibitors, including small molecules and nucleic acid agents, are described. Methods for identification of novel FFA1 inhibitors are also provided.

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 disclosure is directed to systems and methods for treating retinal dystrophy and retinal degenerations. The disclosure describes retina device comprising a first component including an energy conversion and an interface layer comprising gold nanoparticles and a second component including a light-shutter valve (LSV) configured to operate as a neurostimulator.

This invention discloses a kit and method for diagnosing retinal degenerative diseases in ophthalmology, relating to the field of gene detection technology. It includes reaction tubes and detection reagents targeting LincRNA-TUNAR. The core premix contains a thermostable reverse transcriptase and a hot-start DNA polymerase co-immobilized on porous amino resin, supplemented with a PEG-8000 macromolecular crowding reagent and betaine. The signal amplification end includes a CRISPR-Cas12a system based on conditionally activated crRNA. This invention solves the problems of complex secondary structures of lncRNA, extreme susceptibility to endogenous inhibition in micro-samples from ophthalmology, and high false-positive rates in conventional amplification by constructing a solid-phase dual-enzyme spatially confined microreactor and a macromolecular crowding environment, combined with specific dual control using self-locking hairpin crRNA. It exhibits significant advantages such as strong system resilience, support for extraction-free direct amplification of clinical samples, extremely high detection sensitivity, and short diagnostic time.

The present application belongs to the field of animal models, and relates to a kind of RHO-like protein and its application in the construction of retinal pathological animal model.The present application specifically provides a humanized RHO recombinant protein by replacing the amino acid region encoded by the second exon in the Rho protein of mouse with the amino acid region encoded by the second exon in the human RHO protein.Based on the humanized RHO recombinant protein and the humanized RHO recombinant protein with retinal pathogenic mutation, the present application provides a preparation method of humanized mouse model without carrying RHO pathogenic mutation and humanized mouse model carrying RHO pathogenic mutation.The present application provides a preclinical research platform highly simulating human disease for studying the mechanism of RHO pathogenic mutation leading to retinal degeneration and verifying the effectiveness and safety of gene therapy strategy.

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.).