FIELD: gene therapy.
SUBSTANCE: group of inventions relates to gene therapy, in particular to target delivery of transgene-encoding structures to the liver for the expression of therapeutic proteins. Polynucleotide expression structures, and AAV vector containing a polynucleotide expression structure are proposed. A pharmaceutical composition for the treatment of hemophilia in a subject who needs it, and a pharmaceutical composition for the provision of a transgene that encodes a human Factor VIII for a subject who needs it, containing the specified AAV vector are also proposed. A method for the provision of a human Factor VIII for a subject who needs it, and a method for the induction of tolerance in a subject to a human Factor VIII are also proposed.
EFFECT: group of inventions provides for the expression of transgenes at high levels in liver cells, using the specified expression structures.
15 cl, 40 dwg, 9 tbl, 3 ex
| Title | Year | Author | Number |
|---|---|---|---|
| IMPROVEMENT OF CLINICAL PARAMETERS THROUGH FACTOR VIII EXPRESSION | 2019 |
|
RU2799048C2 |
| GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A | 2017 |
|
RU2762257C2 |
| AAV VECTOR COMPOSITIONS AND METHODS FOR GENE TRANSFER TO CELLS, ORGANS AND TISSUES | 2013 |
|
RU2653444C2 |
| CODON-OPTIMIZED NUCLEIC ACID THAT ENCODES B-DOMAIN-DELETED FACTOR VIII PROTEIN AND ITS USE | 2022 |
|
RU2808564C2 |
| CPG REDUCED FACTOR VIII VARIANTS, COMPOSITIONS AND METHODS AND USE FOR TREATMENT OF HEMOSTASIS DISORDERS | 2016 |
|
RU2745506C2 |
| METHODS AND COMPOSITIONS FOR TREATING FABRY DISEASE | 2020 |
|
RU2822369C2 |
| MODIFICATION OF B-CELLS | 2018 |
|
RU2783116C2 |
| METHODS AND COMPOSITIONS FOR THE TREATMENT OF FABRY DISEASE | 2017 |
|
RU2788133C2 |
| RECOVERED NUCLEIC ACID WHICH CODES FVIII-BDD-BASED FUSION PROTEIN AND HETEROLOGOUS SIGNAL PEPTIDE, AND USE THEREOF | 2022 |
|
RU2818229C2 |
| METHODS OF GENOTHERAPY USING FACTOR VIII (FVIII) GENE | 2018 |
|
RU2808274C2 |
