FIELD: biotechnology.
SUBSTANCE: present invention relates to use of genetic constructs containing DNA with closed ends (ceDNA vectors) for expression control. Invention discloses a method for maintaining, stabilizing or increasing the level of expression of a target transgene in a cell or in a subject due to at least one sequential introduction of an additional dose of the ceDNA vector after the initial introduction.
EFFECT: ceDNA vector as disclosed in the present invention can be re-introduced ("repeated dose" or "booster" introduction) to continue expression of the target protein in a subject, wherein the second dose increases the level of expression of the target protein or prolongs it, wherein such repeated administration does not cause an immune response sufficient to prevent achieving expression of the target protein.
35 cl, 11 ex, 6 tbl, 11 dwg
Title | Year | Author | Number |
---|---|---|---|
NON-VIRAL DNA VECTORS AND OPTIONS FOR THEIR USE FOR EXPRESSION OF THERAPEUTIC AGENT BASED ON PHENYLALANINE HYDROXYLASE (PAH) | 2020 |
|
RU2814137C2 |
CLOSED-ENDED DNA VECTORS OBTAINED BY CELL-FREE SYNTHESIS, AND METHOD OF PRODUCING CEDNA VECTORS | 2019 |
|
RU2820586C2 |
NON-VIRAL DNA VECTORS AND THEIR USE FOR THE PRODUCTION OF ANTIBODIES AND FUSION PROTEINS | 2019 |
|
RU2800914C2 |
MODIFIED CLOSED CIRCULAR DNA (CCDNA) CONTAINING SYMMETRIC MODIFIED INVERTED END REPEATED SEQUENCES | 2019 |
|
RU2816963C2 |
NON-VIRAL DNA VECTORS AND OPTIONS FOR THEIR USE FOR EXPRESSION OF THERAPEUTIC AGENT BASED ON FACTOR VIII (FVIII) | 2020 |
|
RU2812852C2 |
MODIFIED CLOSED-ENDED DNA (scDNA) | 2018 |
|
RU2800026C2 |
GENE EDITING USING MODIFIED CLOSED-END DNA (ceDNA) | 2018 |
|
RU2811724C2 |
MODULATION OF REP PROTEIN ACTIVITY WHEN PRODUCING CLOSED-END DNA (ceDNA) | 2020 |
|
RU2812850C2 |
COMPOSITIONS OF NON-VIRAL NON-CAPSID DNA VECTORS BASED ON LIPID NANOPARTICLES | 2018 |
|
RU2778407C2 |
GENE THERAPY VECTORS FOR TREATING DANON'S DISEASE | 2020 |
|
RU2822925C2 |
Authors
Dates
2024-04-08—Published
2019-02-21—Filed