18 results about "Degenerative Disorder" patented technology

Disclosed are devices, systems and methods for restoring spinal alignment and / or maximizing range of motion for patients who suffer from spinal degenerative disorders.

The present disclosure relates generally to alkyne containing pharmaceutical agents, and in particular, to phenylethynyl-thiophene based compounds. More particularly, the present disclosure provides a class of compounds that can inhibit and / or attenuate apoptosis via caspase 3 for the treatment of various degenerative disorders. Additionally, the present disclosure relates to methods for treating specific degenerative disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, epilepsy, spinal cord injury, complication due to diabetes, multiple sclerosis (MS), muscular dystrophy (MD), Parkinson's disease (PD), irritable bowel syndrome (IBS) and Alzheimer's disease (AD) in a patient comprising administering to the patient an effective amount of a present compound.

The present disclosure provides for one or more embodiments for treating retinal degenerative disorders in a subject by administering to the subject an effective amount of allogeneic mesenchymal stem cells to the subject's suprachoroidal area. Allogeneic mesenchymal stem cells, when used in the disclosed treatment regimen, may exhibit anti-inflammatory, immunomodulatory, anti-fibrotic, pro-vascular, and / or pro-regenerative processes. Thus, allogeneic mesenchymal stem cells may be understood to have the potential to target and treat multiple pathological targets implicated in retinal degenerative disorders, including retinal disorders characterized by drusen accumulation, some of which have inherited forms.

The present invention provides methods for treating lysosomal storage disorders such as Gaucher's disease, by using compounds of Formula III as described herein.

Provided are methods for in vitro induced differentiation of embryonic stem cells or pluripotent stem cells into dopaminergic neural progenitor cells, dopaminergic neural precursor cells and / or dopaminergic neurons. Also provided are media for use in the method, cell populations obtained using the method, and the use of the obtained cell populations in the prevention and / or treatment of diseases and / or dysfunctions associated with dopaminergic neuron degenerative disorders.

Provided herein are recombinant expression systems for converting a cell to a retinal photoreceptor induced neuron (PR-iN) that include (a) a nucleic acid sequence encoding a gene cassette comprising one or more of ASCL1, NEUROG2, OTX2, CRX, LHX4, NRL, NR2E3, NEUROD1, IKZF1, IKZF4, MEF2C, MEF2D, MEIS2, ONECUT1, KLF16, SALL3, ZIC3, POU2F1, POU2F2, RORA, RORB, RXRG, or THRB, and (b) a nucleic acid sequence encoding a porcine teschovirus 2A (p2A) peptide sequence. Additionally, the recombinant expression systems disclosed herein can be used to treat retinal degenerative disorders.

The invention provides methods for modulating intestinal immune function, modulating inflammatory responses, treating an inflammatory disorder, treating an autoimmune disorder, treating an allergic disorder, or treating a degenerative disorder in an animal by modulating the amount of one or more phosphatidylglycerol compounds in the animal (e.g., by oral administration of a phosphatidylglycerol compound).

This invention relates to an IRAK4 degrading agent and its uses. This degrading agent can be used to treat or prevent diseases such as cancer, neurodegenerative diseases, viral diseases, autoimmune diseases, inflammatory diseases, hereditary diseases, hormone-related diseases, metabolic diseases, organ transplant-related diseases, immunodeficiency diseases, destructive bone diseases, proliferative diseases, infectious diseases, cell death-related conditions, thrombin-induced platelet aggregation, liver diseases, pathological immune conditions involving T cell activation, cardiovascular diseases, or CNS diseases.

This invention provides a method for producing induced pluripotent stem cells. [Solution] The present disclosure includes the step of producing induced pluripotent stem cells by expressing exogenous nucleic acids encoding proteins OCT3 / 4, SOX2, KLF4, LIN28, and L-MYC, as well as p53-shRNA, in umbilical cord amniotic stem cells under conditions suitable for reprogramming stem cells. The present disclosure also includes the induced pluripotent stem cell population obtainable by this method, the induced pluripotent stem cell population obtained by this method, a pharmaceutical composition containing the induced pluripotent stem cells of the present invention, a method for differentiating the induced pluripotent stem cells of the present invention, a pharmaceutical composition containing the differentiated induced pluripotent stem cells obtained by this method, and a method for treating congenital or acquired degenerative disorders in a subject, comprising the step of administering target cells differentiated from pluripotent stem cells to the subject.

The present application relates to a non-human mammal model of a human neurodegenerative disorder, methods of producing the non-human mammal model, and methods of using the non-human mammal model to identify agents suitable for treating a neurodegenerative disorder. The present application also relates to methods of treating neurodegenerative disorders and restoring normal brain interstitial potassium levels.

A pharmaceutical composition is used for improving health, curing abnormalities and degenerative diseases, and achieving anti-aging therapeutic effect in mammals. The pharmaceutical composition includes a pharmaceutical carrier and an isotope selective component including at least one of a chemical element in which the isotopic ratio of at least one light isotope is altered to exceed natural abundance.

The disclosure provides compounds and method useful for modulating gp130 biological activity. The disclosure also provides methods and compositions for treating disease and disorders associated with gp130 activity, particularly those associated with inflammation.

The invention relates to an IRAK4 degradation agent and application thereof. The degrading agent can be used for treating or preventing cancer, neurodegenerative diseases, viral diseases, autoimmune diseases, inflammatory diseases, hereditary diseases, hormone-related diseases, metabolic diseases, diseases related to organ transplantation, immunodeficiency diseases, destructive bone diseases, proliferative diseases, infectious diseases, conditions related to cell death, and the like. Thrombin-induced platelet aggregation, liver diseases, pathological immune conditions involving T cell activation, cardiovascular diseases or CNS diseases, etc.

The invention discloses a tricyclic compound as well as a preparation method and application thereof. Relates to a compound shown in a general formula (I), a preparation method thereof and a pharmaceutical composition containing the compound. The invention also relates to the application of the compound in preparation of medicines for treating or preventing autoimmune diseases, inflammatory diseases, cancers, viral diseases, neurodegenerative diseases, hereditary diseases, hormone-related diseases, metabolic diseases, organ transplantation-related diseases, immunodeficiency diseases, destructive bone diseases, proliferative diseases, infectious diseases and the like. The invention also relates to the application of the medicine for treating diseases related to cell death or cardiovascular diseases. Wherein each substituent in the general formula (I) is as defined in the specification.

The invention provides a heterocyclic ring substituted pyrazine derivative. The heterocyclic substituted pyrazine derivative has a structure as shown in a formula I. The heterocycle substituted pyrazine derivative disclosed by the invention has an excellent protection effect on cerebral cortex neuronal cells, and has a remarkable inhibition effect on alpha-syn aggregation and an excellent anti-oxidation effect. Therefore, the heterocyclic substituted pyrazine derivative disclosed by the invention can be used as a medicine for preventing and treating nerve injury, degenerative diseases and ischemic diseases.

The present application provides compounds that are PAPD5 inhibitors and are useful in treating a variety of conditions such as cancer, telomere diseases, and aging-related and other degenerative disorders.

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.).

The invention relates to the field of biomedicine, in particular to application of T21 site mutation of PNP protein in treatment of retina degenerative diseases. According to the invention, a CRISPR-Cas9 technology is used to autonomously construct a mutant with a PNP protein T21 site mutated into alanine. Tests prove that the mutant can improve the retina cell purine metabolism homeostasis, regulate and control the retina microenvironment inflammation level and reduce retina cell structure and function damage, can serve as a target spot to research related medicine for treating retina damage, and provides a new thought and means for treatment of retina degenerative diseases.