FIELD: biotechnology.
SUBSTANCE: group of inventions including a recombinant adeno-associated virus (rAAV) for delivering a transgene to liver cells (versions), a composition, a method for delivering a transgene, a method for re-introducing a transgene into a cell for expressing a heterologous gene in a cell, using rAAV for obtaining a drug (variants), a method for treating a subject with a disorder or disease that can be treated by liver expression of a transgene, a method for reintroducing a transgene to express a heterologous gene in a cell to treat a subject with a disorder or disease that can be treated by liver expression of the transgene, a method for treating a subject suffering from the disorder or a disease associated with abnormal activity of an endogenous protein, a vector for producing rAAV (variants), a cell for expressing a heterologous gene, a method for producing rAAV.
EFFECT: invention expands the arsenal of means for delivering the transgene to the liver.
47 cl, 14 dwg, 5 tbl, 2 ex
| Title |
Year |
Author |
Number |
| NEW CAPSID PROTEINS OF ADENO-ASSOCIATED VIRUS |
2017 |
- Colosi, Peter, Cameron
- Lochrie, Michael
- Ng, Robert
|
RU2779318C2 |
| RECOMBINANT VIRAL PARTICLES WITH MODIFIED TROPISM AND WAYS OF THEIR USE FOR TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS |
2018 |
- Sabin, Lea
- Shonerr, Kristofer
- Ekonomides, Aris N.
- Kiratsus, Kristos
- Merfi, Endryu Dzh.
|
RU2811426C2 |
| VERSIONS BASED ON ADENO-ASSOCIATED VIRUS, COMPOSITIONS AND METHODS FOR PULMONARY DELIVERY |
2021 |
- Kotterman, Melissa
- Francis, Peter
- Calton, Melissa
- Gonzales, Johnny
- Croze, Roxanne
- Schmitt, Christopher
|
RU2829874C1 |
| RECOMBINANT VIRAL VECTORS WITH MODIFIED TROPISM AND WAYS OF THEIR USE FOR TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS |
2018 |
- Kiratsus, Kristos
- Merfi, Endryu Dzh.
- Vang, Cheng
- Sabin, Lea
|
RU2809246C2 |
| COMPOSITIONS AND METHODS FOR INCREASING OR ENHANCING TRANSDUCTION OF GENE THERAPY VECTORS AND FOR REMOVING OR REDUCING IMMUNOGLOBULINS |
2021 |
|
RU2841237C1 |
| 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 |
| VERSION OF AAV, COMPOSITIONS AND METHODS IN WHICH IT IS USED, AS WELL AS METHODS OF USING IT FOR TRANSFERRING GENES INTO CELLS, ORGANS AND TISSUES |
2014 |
- High, Katherine A.
- Yazicioglu, Mustafa N.
- Anguela, Xavier
|
RU2837753C2 |
| ISOLATED MODIFIED VPI CAPSID PROTEIN OF ADENO-ASSOCIATED VIRUS OF SEROTYPE 9 (AAV9), CAPSID AND VECTOR BASED THEREON |
2021 |
- Strelkova Anna Nikolaevna
- Shugaeva Tatyana Evgenevna
- Gershovich Pavel Mikhajlovich
- Yakovlev Pavel Andreevich
- Morozov Dmitrij Valentinovich
|
RU2825667C2 |
| GENOTHERAPY CONSTRUCTIONS FOR TREATMENT OF WILSON'S DISEASE |
2020 |
- Livingston, Christine
- Wadsworth, Samuel
|
RU2807158C2 |
| ISOLATED MODIFIED VP1 CAPSID PROTEIN OF ADENO-ASSOCIATED VIRUS OF SEROTYPE 5 (AAV5), CAPSID AND VECTOR BASED ON IT |
2019 |
- Strelkova Anna Nikolaevna
- Karabelskij Aleksandr Vladimirovich
- Madera Dmitrij Aleksandrovich
- Perepelkina Mariya Pavlovna
- Yurlova Elena Viktorovna
- Gershovich Pavel Mikhajlovich
- Prokofev Aleksandr Vladimirovich
- Morozov Dmitrij Valentinovich
|
RU2751592C2 |
