A61K38/44

REGULATION OF GENE EXPRESSION BY APTAMER-MEDIATED MODULATION OF ALTERNATIVE SPLICING

The invention provides a platform and methods of using the platform for the regulation of the expression of a target gene using exposure to an aptamer ligand (for example, a small molecule). The platform features a polynucleotide gene regulation cassette that is placed in the target gene and includes a synthetic riboswitch positioned in the context of a 5′ intron-alternative exon-3′ intron. The riboswitch comprises an effector region and a sensor region (e.g., an aptamer that binds a small molecule ligand) such that the alternative exon is spliced into the target gene mRNA when the ligand is not present thereby preventing expression of the target gene. When the ligand is present, the alternative exon is not spliced into the target gene mRNA thereby providing expression of the target gene.

CELLS WITH INCREASED IMMUNO-REGULATORY PROPERTIES AND METHODS FOR THEIR USE AND MANUFACTURE

The present invention is directed to compositions and methods to increase the expression of PD-L1 and/or IDO-1 in a population of cells, the modulated cells expressing increased PD-L1 and/or IDO-1, and methods related to the immunosuppressive effects obtained by cells expressing increased PD-L1 and/or IDO-1.

CELLS WITH INCREASED IMMUNO-REGULATORY PROPERTIES AND METHODS FOR THEIR USE AND MANUFACTURE

The present invention is directed to compositions and methods to increase the expression of PD-L1 and/or IDO-1 in a population of cells, the modulated cells expressing increased PD-L1 and/or IDO-1, and methods related to the immunosuppressive effects obtained by cells expressing increased PD-L1 and/or IDO-1.

PHENYLALANINE HYDROXYLASE VARIANTS AND USES THEREOF
20230235298 · 2023-07-27 ·

Variant phenylalanine hydroxylase polypeptides having substitutions at selected amino acid residues are disclosed. Also disclosed are methods of using variant phenylalanine hydroxylase polypeptides, or polynucleotides encoding variant phenylalanine hydroxylase polypeptides, to treat disorders such as phenylketonuria.

SELF-ASSEMBLED CATALASE NANOPARTICLE AND PREPARATION METHOD THEREFOR AND USE THEREOF

Disclosed are a self-assembled catalase nanoparticle and a preparation method therefor and the use thereof. The self-assembled catalase nanoparticle of the present invention is obtained by dissolving catalase freeze-dried powder to obtain a catalase solution, adjusting the pH value of the catalase solution, and then centrifugating or filtering same to obtain a supernatant or a filtrate, and further thermally incubating the supernatant or filtrate. The self-assembling catalase nanoparticle of the present invention can be used in medicines or food products that promote immune cell growth and regulate organic immunity.

SELF-ASSEMBLED CATALASE NANOPARTICLE AND PREPARATION METHOD THEREFOR AND USE THEREOF

Disclosed are a self-assembled catalase nanoparticle and a preparation method therefor and the use thereof. The self-assembled catalase nanoparticle of the present invention is obtained by dissolving catalase freeze-dried powder to obtain a catalase solution, adjusting the pH value of the catalase solution, and then centrifugating or filtering same to obtain a supernatant or a filtrate, and further thermally incubating the supernatant or filtrate. The self-assembling catalase nanoparticle of the present invention can be used in medicines or food products that promote immune cell growth and regulate organic immunity.

METHODS AND KITS FOR PREDICTING INFUSION REACTION RISK AND ANTIBODY-MEDIATED LOSS OF RESPONSE BY MONITORING SERUM URIC ACID DURING PEGYLATED URICASE THERAPY
20230028134 · 2023-01-26 ·

Methods and kits for predicting infusion reaction risk and antibody-mediated loss of response during intravenous PEGylated uricase therapy in gout patients is provided. Routine SUA monitoring can be used to identify patients receiving PEGylated uricase who may no longer benefit from treatment and who are at greater risk for infusion reactions.

METHODS AND KITS FOR PREDICTING INFUSION REACTION RISK AND ANTIBODY-MEDIATED LOSS OF RESPONSE BY MONITORING SERUM URIC ACID DURING PEGYLATED URICASE THERAPY
20230028134 · 2023-01-26 ·

Methods and kits for predicting infusion reaction risk and antibody-mediated loss of response during intravenous PEGylated uricase therapy in gout patients is provided. Routine SUA monitoring can be used to identify patients receiving PEGylated uricase who may no longer benefit from treatment and who are at greater risk for infusion reactions.

Vitamins and Alpha Keto Acid Compositions for Use in a Treatment Program for Chronic Kidney Disease Patients
20230028626 · 2023-01-26 · ·

A vitamin for use by kidney patients in Stages 3, 4, 4a, 4b, and 5, who are not on dialysis, comprising administering daily a: low nitrogen diet; low nitrogen protein food comprising magnesium and/or calcium salts of alpha keto acids; and time released vitamin. The vitamin comprises the active ingredients of: Zinc; Selenium; Vitamins B1, B2, B6, B12, B5, E, and K2; Niacin; superoxide dismutase; catalase; glutathione and Folate; and excludes calcium, magnesium, phosphorus, sodium, manganese, fluoride, Vitamin D and K1. The low nitrogen food contains daily up to 300 mg of nitrogen; and at least five alpha keto analogues of magnesium and/or calcium salts of: leucine, isoleucine, methionine (MEMS-II), phenylalanine (PAMS-I), and valine. Methods of making and the chemical structures MEMS-II and the PAMS-I are disclosed. The treatment results in an increase of Glomerular Filtration Rate (GFR), a decrease in blood urea levels, and a decrease in creatinine levels.

Vitamins and Alpha Keto Acid Compositions for Use in a Treatment Program for Chronic Kidney Disease Patients

A low nitrogen protein food composition Albutrix™ for use by kidney patients in Stages 3, 4, 4a, 4b, and 5, who are not on dialysis, in a treatment program comprising administering daily a: low nitrogen diet; low nitrogen protein food comprising magnesium and/or calcium salts of alpha keto acids; and time released vitamin. The vitamin comprises the active ingredients of: Zinc; Selenium; Vitamins B1, B2, B6, B12, B5, E, and K2; Niacin; superoxide dismutase; catalase; glutathione and Folate; and excludes calcium, magnesium, phosphorus, sodium, manganese, fluoride, Vitamin D and K1. The low nitrogen food contains daily up to 300 mg of nitrogen; and at least five alpha keto analogues of magnesium and/or calcium salts of: leucine, isoleucine, methionine (MEMS-II), phenylalanine (PAMS-I), and valine. The treatment results in an increase of Glomerular Filtration Rate (GFR), a decrease in blood urea levels, and a decrease in creatinine levels.