58 results about "Retinitis" 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.

Provided herein is a method of treating a condition associated with nuclear speckle sphericity in a patient, including administering to the patient an amount of a composition including a nuclear speckle rejuvenator effective to reduce nuclear speckle sphericity in the patient.

To provide Akt3 modulators and compounds and pharmaceutical compositions for treating diseases by modulation of Akt3 signaling.SOLUTION: A compound of Formula Ib or Ic, or a pharmaceutically acceptable salt thereof, and a pharmaceutical composition comprising said compound for treating a disease in a subject in need thereof, A pharmaceutical composition wherein the treatment comprises administering to a subject an effective amount of the compound; optionally wherein the disease is selected from the group consisting of neurodegenerative diseases, cachexia, eating disorders, obesity, complications of obesity, inflammatory diseases, virally-induced inflammatory reactions, Gulf War Syndrome, tuberous sclerosis, retinitis pigmentosa, graft rejection, cancer, autoimmune diseases, ischemic tissue injury, traumatic tissue injury and combinations thereof.SELECTED DRAWING: Figure 6

Described herein are methods and devices for the long term treatment of ophthalmic disorders. Also disclosed are encapsulated cell therapy (ECT) devices that secrete a biologically active molecule and methods for using the same for the treatment of various kinds of ophthalmic disorders, including retinitis pigmentosa, geographic atrophy (dry age-related macular degeneration), glaucoma and / or macular telangiectasia.

Pyrvinium compounds, or a pharmaceutically acceptable salt thereof, used in methods for reducing tauopathy in a subject in need thereof. Pyrvinium compounds, or a pharmaceutically acceptable salt thereof, used in methods for treating a disease associated with accumulation of misfolded proteins (e.g., proteinopathies, Alzheimer's disease, frontotemporal dementia, Parkinson's disease, retinitis pigmentosa such as retinitis pigmentosa with rhodopsin mutations, aging, neurodegenerative diseases, or metabolic disorders).

An object of the present invention is to provide a composition for preventing visual dysfunction caused by exposure to light, such as so-called blue light. The present invention provides a composition comprising a lactic acid bacterium as an active ingredient for use in suppressing or improving conditions caused by retinal inflammation resulting from light damage. Eye discomfort is an exemplary condition caused by retinal inflammation resulting from light damage.

CRISPR / Cas-related compositions and methods for treatment of Usher Syndrome and / or Retinitis Pigmentosa are disclosed herein.

Benzyl derivative compounds having peroxisome proliferator-activated receptor alpha (PPARα) agonist activity, kits and compositions containing such compounds, and methods of using them to enhance PPARα activity to treat diseases and / or conditions involving inflammation and / or angiogenesis, particularly diseases and / or conditions of the eye such as, but not limited to, retinal inflammation, retinal neovascularization, retinal vascular leakage, retinopathy of prematurity, diabetic retinopathy, age-related macular degeneration, and diabetic macular edema.

Compositions and methods for detecting genetic structural variations (SVs) in a gene of interest, involved in disease pathologies commonly found in large Mendelian genomics programs; or for genetic mapping of mobile transposable elements, involved in genetic disease, cancer, and aging are disclosed. The methods include: (i) contacting a sample comprising genomic DNA with a DNA endonuclease for an effective amount of time to cleave the genomic DNA into fragments, the genomic DNA has not been crosslinked, (ii) subjecting the fragments obtained from step (b) to a DNA ligase to obtain circularized DNA, (iii) subjecting the circularized DNA to inverse PCR amplification comprising inverse primers, wherein the inverse primers are designed to align with anticipated wild-type sequences, adjacent to a suspected mutation locus in a gene, and (iv) sequencing the amplified product. Exemplary conditions include Bardet-Biedl syndrome; Severe upper and lower limb defects; Retinitis pigmentosa; Syndromic microcephaly; Spastic paraplegia; and atypical hemolytic uremic syndrome.

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 application provides application of thrombospondin 1 in preparation of a drug for preventing and / or treating retinal injury, and belongs to the technical field of eye disease drugs. The application verifies the anti-inflammatory effect of thrombospondin 1 (THBS-1) in experimental blue light induced retinal inflammation by means of in vitro cell experiments and in vivo animal experiments, and verifies the role of thrombospondin 1 in inhibiting pathological neovascularization and the protective effect on retinal injury. Therefore, the application provides application of thrombospondin 1 in preparation of a drug for preventing and / or treating age-related macular degeneration.

Benzyl derivative compounds having peroxisome proliferator-activated receptor α (PPARα) agonistic activity, kits and compositions containing such compounds, and methods of their use in enhancing PPARα activity for treating diseases and / or conditions involving inflammation and / or angiogenesis, particularly ocular diseases and / or conditions such as but not limited to retinal inflammation, retinal neovascularization, retinal vascular leakage, retinopathy of prematurity, diabetic retinopathy, age-related macular degeneration, and diabetic macular edema.

A microorganism containing Bacteroides fragilis genes is used as an active ingredient in the preparation of pharmaceuticals, medical devices, or food for treating optic nerve damage. This delivers Bacteroides fragilis (or its functional components) into the gastrointestinal tract, which helps maintain the number of retinal ganglion cells and the thickness of the retinal nerve fiber layer, inhibits the activation of retinal microglia, inhibits the activation of retinal inflammatory pathways and the expression of inflammatory mediators, thereby protecting visual function.

The invention relates to a compound indoleacetic acid and application of the compound indoleacetic acid as an active ingredient in preparation of medicines or foods for treating optic nerve injury, and the indoleacetic acid is given in vivo, so that the number of retinal ganglion cells and the thickness of a retinal nerve fiber layer are favorably maintained, and activation of retinal microglial cells is inhibited; retina inflammation pathway activation and inflammatory mediator expression are inhibited, so that the visual function is protected.

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

The microorganism contains the bacteroides fragilis gene, and the bacteroides fragilis gene is used as an active ingredient to be applied to preparation of medicine or food for treating optic nerve damage, the bacteroides fragilis (or functional components thereof) is delivered to gastrointestinal tracts, the number of retinal ganglion cells and the thickness of a retinal nerve fiber layer can be maintained, activation of retinal microglia cells is inhibited, and optic nerve damage is inhibited. Retina inflammation pathway activation and inflammatory mediator expression are inhibited, so that the visual function is protected.

The application relates to a compound indole acetic acid, and application of the compound indole acetic acid as an active ingredient in the preparation of a medicament or food for treating optic nerve damage. In vivo administration of the indole acetic acid is beneficial to maintaining the number of retinal ganglion cells and the thickness of the retinal nerve fiber layer, inhibiting the activation of retinal microglia cells, inhibiting the activation of a retinal inflammation channel and the expression of inflammatory mediators, and protecting visual function.

The invention provides compositions and methods for ameliorating defects in myosin VIIa (MYO7A) expression and / or function, including providing vectors for myosin VIIa expression and formulations comprising them, and methods of using them, for treating human retinitis pigmentosa (or retinal degeneration), and blindness and deafness such as that found in Usher syndrome. The invention provides in vivo gene therapy for ameliorating defects in myosin VIIa (MYO7A) expression and / or function, including compositions and methods for gene transfer of a human myosin VIIa (MYO7A) polynucleotide sequence that expresses multiple polypeptide isoforms of human myosin VIIa.

Methods for facilitating the genesis of photoreceptor rods in a subject by administering a therapeutically effective amount of a therapeutic agent are provided. Other methods are provided for treating retinitis pigmentosa by administering a therapeutically effective dose of therapeutic agents. Methods for producing an in vitro population of progenitor cells and photoreceptor rods generated therefrom are also provided.

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).

Methods and compositions for the use of CRISPR / Cas9 technology for treating RP1 mutation-associated autosomal dominant Retinitis Pigmentosa (adRP).

The invention relates to the field of biological medicines, in particular to a nano-drug for treating eye diseases and a preparation method and application thereof. According to the invention, myricetin is wrapped by a polymer PLGA-PEG-HA, so that the prepared nano-drug prolongs the residence time of the drug in eyes, the release cycle and bioavailability of the drug are obviously improved, and the nano-drug has a long-acting protective effect on optic nerve injury and retinal inflammation and is remarkable in curative effect; the hyaluronic acid modified nanoparticles can improve the biocompatibility and inflammation targeting ability of the drug. A suspension obtained after the nano-drug is redissolved can be injected into a vitreous body through a microsyringe, and a long-acting protection or treatment strategy is provided for treatment of optic nerve injury and retinal inflammation.

The present invention relates to methods and compositions for gene therapy of retinitis pigmentosa related to mutations in pre-mRNA processing factor 31 (PRPF31).

The presently disclosed subject matter is directed to compositions and methods for treating CaMKK2-mediated ophthalmic diseases, including but not limited to 1) ocular surface inflammatory diseases (OSIDs), including but not limited to ocular graft versus host disease, ocular cicatricial pemphigoid, vernal keratoconjunctivitis, allergic eye disease, meibomian gland dysfunction, aqueous tear deficiency (common dry eye disease), corneal scarring, and conjunctival scarring and fibrosis; 2) uveitis and other inflammatory diseases of the eye, including but not limited to keratitis, scleritis, iritis, iridocyclitis, intermediate uveitis, pars planitis, posterior uveitis, choroiditis, chorioretinitis, retinitis, or panuveitis of noninfectious, infectious, or idiopathic etiologies; and 3) “back of the eye” retinal diseases, which include dry age-related macular degeneration, neovascular age-related macular degeneration, diabetic retinopathy, retinal vascular diseases (e.g. retinal vein occlusion, retinal artery occlusion), and retinal degenerations and dystrophies, in a subject. Particularly, the disclosed compounds exhibit improvements over STO-609, a well characterized specific inhibitor of CaMKK2. The disclosed compounds exhibit enhanced aqueous solubility and formulation, as well as elimination of non-binding isomers during production. The disclosed inhibitor compounds can be used to effectively treat ophthalmic diseases, cancers, appetite disorders, systemic inflammatory diseases, and the like.

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.

Benzyl derivative compounds having peroxisome proliferator-activated receptor alpha (PPAR alpha) agonistic activity, kits and compositions comprising such compounds, and methods of their use to enhance PPAR alpha activity for the treatment of diseases and / or disorders involving inflammation and / or angiogenesis, in particular ocular diseases and / or disorders, such as inflammatory diseases and / or angiogenesis. Such as, but not limited to, retinal inflammation, retinal neovascularization, retinal vascular leakage, preterm retinopathy, diabetic retinopathy, age-related macular degeneration, and diabetic macular edema.

The present invention is directed vectors comprising a FCBR1-F0.4 promotor or an IRBP-GRK1 promotor and a nucleic acid sequence encoding a ciliary protein, or fragment or variant thereof. In particular, the invention relates to a method of treatment and prevention of retinal ciliopathies, such as retinitis pigmentosa 28.

Disclosed are recombinant meganucleases engineered to recognize and cleave recognition sequences present in a mutant RHO P23H allele. The invention further relates to the use of such recombinant meganucleases in methods for treating retinitis pigmentosa, wherein the mutant RHO P23H allele is preferentially targeted, cleaved, and inactivated.

The present disclosure relates to the technical field of biomedicine, and in particular to use of miRNA or modified miRNA in treating fundus oculi diseases. The miRNA or modified miRNA has biological activity, can be absorbed by cells in vitro or retina cells in vivo, and quickly plays a biological role. Through a topical administration mode, the miRNA or the modified miRNA can inhibit pathological neovascularization, promote recovery of a non-perfusion region, reduce expression of retinal inflammatory factors, relieve retinal fibrosis, and promote nerve injury repair. The method has very good application and research values in the field of biomedicine.