FIELD: pharmaceuticals; medicine.
SUBSTANCE: group of inventions relates to methods for increasing the level of factor VIII (FVIII) protein in a person with hemophilia A, including intravenous administration to a person of one or more doses from 5×1012 to 5×1013 vg/kg adenovirus-associated virus (AAV) vector that encodes the FVIII protein.
EFFECT: providing a person with hemophilia A with an increase in the level of FVIII or normal levels of FVIII.
12 cl, 10 dwg, 2 tbl, 7 ex
| Title | Year | Author | Number |
|---|---|---|---|
| CPG REDUCED FACTOR VIII VARIANTS, COMPOSITIONS AND METHODS AND USE FOR TREATMENT OF HEMOSTASIS DISORDERS | 2016 |
|
RU2745506C2 |
| METHODS OF GENOTHERAPY USING FACTOR VIII (FVIII) GENE | 2018 |
|
RU2808274C2 |
| GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A | 2017 |
|
RU2762257C2 |
| RECOVERED NUCLEIC ACID WHICH CODES FVIII-BDD-BASED FUSION PROTEIN AND HETEROLOGOUS SIGNAL PEPTIDE, AND USE THEREOF | 2022 |
|
RU2818229C2 |
| GENOTHERAPY CONSTRUCTIONS FOR TREATMENT OF WILSON'S DISEASE | 2020 |
|
RU2807158C2 |
| MODIFIED FACTOR IX, AS WELL AS COMPOSITIONS, METHODS AND OPTIONS FOR THE USE OF GENE TRANSFER INTO CELLS, ORGANS AND TISSUE | 2016 |
|
RU2811445C2 |
| OPTIMIZED GENES AND EXPRESSION CASSETTES OF MINI-DYSTROPHIN AND THEIR APPLICATION | 2017 |
|
RU2753194C2 |
| CODON-OPTIMIZED NUCLEIC ACID THAT ENCODES B-DOMAIN-DELETED FACTOR VIII PROTEIN AND ITS USE | 2022 |
|
RU2808564C2 |
| NUCLEIC ACID MOLECULES AND WAYS OF USING THEM | 2018 |
|
RU2819144C2 |
| NUCLEIC ACID CONSTRUCTIONS AND VECTORS FOR GENOTHERAPY FOR TREATING WILSON'S DISEASE AND OTHER CONDITIONS | 2015 |
|
RU2745567C2 |
