FIELD: biotechnology.
SUBSTANCE: described is a group of inventions involving a method for increasing the level of expression of a transgene in tissue and a recombinant viral vector for use in treating methylmalonic acidemia (MMA). In one embodiment, the method involves a step of administering to an individual in need thereof a composition which delivers a transgene which is integrated into the genome of at least a cell population in the individual’s tissue.
EFFECT: invention extends the range of agents for enhancing the expression of the transgene in tissue.
18 cl, 32 dwg, 4 tbl, 18 ex
| Title |
Year |
Author |
Number |
| METHODS FOR TREATMENT OF DANON'S DISEASE AND OTHER AUTOPHAGY DISORDERS |
2017 |
- Adler, Eric, D.
- Nelson, Bradley
- Hashem, Sherin
|
RU2777571C2 |
| COMPOSITIONS AND METHODS FOR DEGRADATION OF IMPROPERLY PACKAGED PROTEINS |
2016 |
|
RU2761564C2 |
| GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A |
2017 |
- Wang, Lili
- Wilson, James M.
- Sidrane, Jenny Agnes
|
RU2762257C2 |
| AAV VECTORS AIMED AT THE CENTRAL NERVOUS SYSTEM |
2015 |
|
RU2727015C2 |
| DNA ANTIBODY CONSTRUCTIONS FOR USE AGAINST LYME DISEASE |
2017 |
- Weiner, David, B.
- Flingai, Seleeke
|
RU2813829C2 |
| TREATMENT OF COMPLEMENT-MEDIATED DISORDERS |
2017 |
- Lachmann, Peter
- Alexander, Ian
|
RU2768982C2 |
| MODIFIED FACTOR IX, AS WELL AS COMPOSITIONS, METHODS AND OPTIONS FOR THE USE OF GENE TRANSFER INTO CELLS, ORGANS AND TISSUE |
2016 |
- Khaj Ketrin A.
- Angela Khaver
|
RU2811445C2 |
| COMPOSITIONS AND METHODS OF GENE EDITING |
2019 |
- Chen, Jin
- Gilbert, Luke
- Nunez, James
- Weissman, Jonathan
|
RU2804665C2 |
| GLYCAN-DEPENDENT IMMUNOTHERAPY MOLECULES |
2016 |
- Demetriou Majkl
- Chzhou Rejmond Venkhou
|
RU2754661C2 |
| THERAPEUTIC AGENTS AND METHODS FOR THE TREATMENT OF TLR2-MEDIATED DISEASES AND DISORDERS |
2019 |
- Witztum, Joseph, L.
- Tsimikas, Sotirios
- Que, Xuchu
|
RU2791022C2 |
