A61K38/1709

COMPOSITIONS AND METHODS OF INCREASING LONGEVITY OR TREATING CELLULAR STRESS
20180008679 · 2018-01-11 ·

The present invention includes methods and compositions for increasing longevity of a cell and increasing cellular resistance to stress. In certain embodiments, the invention includes a method to induce gene expression of a homolog of Pachytene Checkpoint 2 (pch-2) or bmk-1 gene. The present invention also includes methods to treat oxidative stress or induce cellular death or apoptosis by administering a composition comprising a modulator of pch-2 or bmk-1 homolog gene expression.

MODIFIED LIGAND-GATED ION CHANNELS AND METHODS OF USE
20180009862 · 2018-01-11 ·

This document relates to materials and methods for controlling ligand gated ion channel (LGIC) activity. For example, modified LGICs including at least one LGIC subunit having a modified ligand binding domain (LBD) and/or a modified ion pore domain (IPD) are provided. Also provided are exogenous LGIC ligands that can bind to and activate the modified LGIC, as well as methods of modulating ion transport across the membrane of a cell of a mammal, methods of modulating the excitability of a cell in a mammal, and methods of treating a mammal having a channelopathy.

Arginine-rich Peptide Mixture, their Application Thereof in Cervical Cancer Therapy, and a Process for Producing same
20180009842 · 2018-01-11 ·

A process for producing an arginine-rich peptide mixture and the application thereof in cervical cancer therapy is provided. The process includes the following steps: A suspension of walnut meal and egg albumin is pretreated with ultrahigh pressure, and then digested by alkaline proteinase and papain in separated steps with the ultrasonic and microwave-assisted extraction. The peptides of interest are isolated from filtration supernatant obtained after the enzyme digestion by reversed phase high-performance liquid chromatography. By using the peptide mixture as a template, acrylic acid and methyl acrylic acid as functional monomers, triethylene glycol dimethacrylate as cross-linking agent, and isopropylthioxanthone in acetone as a photoinitiator, polymerization is induced by ultraviolet light to form a surface imprinted membrane for isolating and enriching the peptides of interest from the supernatant. The arginine content in the peptide mixture is more than 18%. The arginine-rich peptide mixture is able to strongly suppress the proliferation of human cervical cancer Hela cells. The approach is applicable to reduce the cost of production and speed up the commercialization of large-scale production.

Polymer hydrogels for in vivo applications and methods for using and preparing same

Compositions and methods are described for a polymer hydrogel created by a cycloaddition reaction between an azide and an alkyne that proceeds rapidly without catalyst to produce the polymer hydrogel in less than ninety seconds. The polymer hydrogel can be used in in vivo applications for the localized delivery of therapeutic agent in aqueous solutions. An example of therapeutic delivery of a protein in a mouse model is demonstrated.

EFFECTIVE TREATMENTS FOR VASCULAR ASSOCIATED MACULOPATHY, SEVERE MACULOPATHY, LATE-STAGE MACULOPATHY, AND ABERRANT CHORIOCAPILLARIS

Disclosed herein are methods and compositions for the diagnosis and treatment of Vascular Associated Maculopathy, or a symptom thereof, in a subject. Disclosed herein are methods and compositions for the diagnosis and treatment of one or more symptoms associated with Vascular Associated Maculopathy Disclosed in a subject. Disclosed herein are methods and compositions for the diagnosis and treatment of severe maculopathy or last stage maculopathy in a subject. Disclosed herein are methods and compositions for the diagnosis and treatment of resolving aberrant choriocapillaris lobules in a subject.

PRODUCTION OF RECOMBINANT LUBRICIN
20230234999 · 2023-07-27 ·

Disclosed are new recombinant isoforms of human-like lubricin or PRG4 glycoprotein having outstanding lubrication properties and a novel glycosylation pattern, and methods for their manufacture at high levels enabling commercial production.

METHOD OF TREATMENT OF CONGENITAL MYASTHENIC SYNDROME USING DOK7 GENE OR POLYPEPTIDE
20230233646 · 2023-07-27 ·

The present invention relates to methods of preventing or treating a congenital myasthenic syndrome (CMS) in a subject, wherein the CMS is (a) congenital myasthenic syndrome associated with AChR deficiency or (b) fast-channel congenital myasthenic syndrome (FCCMS), the method comprising administering an effective amount of a DOK7 gene or a Dok-7 polypeptide, preferably a rAAV-DOK7 vector, to a subject in need thereof. The invention also relates to products for use in such methods.

METHOD OF TREATMENT OF CONGENITAL MYASTHENIC SYNDROME USING DOK7 GENE OR POLYPEPTIDE
20230233646 · 2023-07-27 ·

The present invention relates to methods of preventing or treating a congenital myasthenic syndrome (CMS) in a subject, wherein the CMS is (a) congenital myasthenic syndrome associated with AChR deficiency or (b) fast-channel congenital myasthenic syndrome (FCCMS), the method comprising administering an effective amount of a DOK7 gene or a Dok-7 polypeptide, preferably a rAAV-DOK7 vector, to a subject in need thereof. The invention also relates to products for use in such methods.

Adeno-Associated Virus Vector Delivery of Microrna-29 to Treat Muscular Dystrophy

The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.

Dry Powder Formulations for Messenger RNA

The present invention provides stable, dry powder messenger RNA formulations for therapeutic use, and methods of making and using the same.