FIELD: biotechnology.
SUBSTANCE: following is described: a group of inventions including a capsid-free vector based on closed-ended DNA (ceDNA) for the expression of factor VIII (FVIII) protein, a method of expressing the FVIII protein in a cell, a pharmaceutical composition for gene therapy, a method of treating a subject suffering from hemophilia A, a cell for producing the protein FVIII, a ceDNA vector delivery composition and a ceDNA vector delivery kit. In one embodiment, the capsidless vector comprises at least one nucleic acid sequence located between flanking inverted terminal repeats (ITRs), wherein the at least one nucleic acid sequence encodes at least one factor VIII (FVIII) protein and is codon optimized for expression in the cell.
EFFECT: invention expands the range of agents for expressing factor VIII (FVIII) protein.
53 cl, 16 dwg, 15 tbl, 14 ex
| Title | Year | Author | Number |
|---|---|---|---|
| MODULATION OF REP PROTEIN ACTIVITY WHEN PRODUCING CLOSED-END DNA (ceDNA) | 2020 |
|
RU2812850C2 |
| NUCLEIC ACID MOLECULES AND WAYS OF USING THEM | 2018 |
|
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| GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A | 2017 |
|
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| RECOVERED NUCLEIC ACID WHICH CODES FVIII-BDD-BASED FUSION PROTEIN AND HETEROLOGOUS SIGNAL PEPTIDE, AND USE THEREOF | 2022 |
|
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|
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| IMPROVEMENT OF CLINICAL PARAMETERS THROUGH FACTOR VIII EXPRESSION | 2019 |
|
RU2799048C2 |
| ANTIBODY WHICH IS CAPABLE OF NEUTRALIZING A SUBSTANCE HAVING AN ACTIVITY OF AN ALTERNATIVE COAGULATION FACTOR FUNCTION VIII (FVIII) | 2015 |
|
RU2737145C2 |
| METHOD FOR MEASURING REACTIVITY OF FVIII | 2015 |
|
RU2752595C2 |
| HIGH GLYCATED FUSED PROTEIN BASED ON HUMAN BLOOD COAGULATION FACTOR VIII, METHOD FOR PRODUCTION THEREOF AND USE THEREOF | 2016 |
|
RU2722374C1 |
| AAV5-BASED VACCINE FOR INDUCTION OF SPECIFIC IMMUNITY TO SARS-COV-2 VIRUS AND/OR PREVENTION OF CORONAVIRUS INFECTION CAUSED BY SARS-COV-2 | 2020 |
|
RU2760301C1 |
