FIELD: biotechnology.
SUBSTANCE: disclosed is a method for preventing, treating or relieving a complement-mediated disorder associated with C3b complement feedback cycle hyperactivity in a patient, and a pharmaceutical composition and a kit for implementing said method are also disclosed. Also described are recombinant viral vectors encoding factor I, recombinant viral particles encapsulating these vectors, and their use in methods of treatment.
EFFECT: invention provides a significant increase in the level of factor I in plasma, which leads to achieving a systemic increase in the level of C3b-inactivating and iC3b-degrading activity in the patient.
44 cl, 9 dwg, 2 tbl, 2 ex
| Title |
Year |
Author |
Number |
| GENE THERAPY |
2016 |
- Grundal, Kristian
- Fannell, Tim
- Khollovud, Kris
|
RU2740038C2 |
| NEW LIVER TARGETING ADENO-ASSOCIATED VIRAL VECTORS |
2019 |
- Colosi, Peter Cameron
- Ramirez, Silvia
|
RU2793735C2 |
| METHODS FOR AAV DETECTION |
2017 |
- Jin, Xiaoying
- O'Riordan, Catherine, R.
- Liu, Lin
- Zhang, Kate
|
RU2771622C2 |
| 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 |
| GENOTHERAPY CONSTRUCTIONS FOR TREATMENT OF WILSON'S DISEASE |
2020 |
- Livingston, Christine
- Wadsworth, Samuel
|
RU2807158C2 |
| GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A |
2017 |
- Wang, Lili
- Wilson, James M.
- Sidrane, Jenny Agnes
|
RU2762257C2 |
| NUCLEIC ACID CONSTRUCTIONS AND VECTORS FOR GENOTHERAPY FOR TREATING WILSON'S DISEASE AND OTHER CONDITIONS |
2015 |
- Murillo Sauca, Oihana
- Gonzalez Aseguinolaza, Gloria
- Hernandez Alcoceba, Ruben
|
RU2745567C2 |
| 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 |
| ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MUCOLIPIDOSIS TYPE II |
2016 |
- Jyu, Nelson
- Dzin, Dong-Kiu
|
RU2742612C2 |
| RECOMBINANT AAV VECTORS EXPRESSING OSTEOPROTECTIVE GENES, INCLUDING HAS2 AND LUBRICIN, SUITABLE IN TREATMENT OF OSTEOARTHRITIS AND SIMILAR JOINT DISEASES IN MAMMALS |
2017 |
- Dias Figejredo Monika
- Kuostio-Mor Sirka Rm
- Bertelet Patrisiya
|
RU2771490C2 |
