FIELD: biotechnology.
SUBSTANCE: compositions and methods for the treatment of ophthalmic disorders in a subject are described. A vector based on an adeno associated virus that includes a nucleic acid molecule containing a sequence that encodes CNGA3, a vector based on an adeno associated virus that includes a nucleic acid molecule containing a sequence that encodes CNGB3, and a vector based on an adeno associated virus that includes a nucleic acid molecule containing a sequence that encodes REP-1 are also described.
EFFECT: invention expands the arsenal of means for the treatment of ophthalmic diseases.
39 cl, 12 dwg, 23 tbl, 7 ex
| Title |
Year |
Author |
Number |
| METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISORDERS AND DISEASES RELATED TO RDH12 |
2017 |
- Bennett, Dzhin
- Sun, Tszyunvej
- Vasireddi, Vidiullata
|
RU2764920C2 |
| INTRATHECAL ADMINISTRATION OF VECTORS BASED ON ADENO ASSOCIATED VIRUSES FOR GENE THERAPY |
2016 |
- Wilson James M.
- Hinderer Christian
- Rothwell William Thomas
|
RU2775138C2 |
| THERAPEUTIC COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE |
2015 |
- Dejvidson Beverli L.
- Mas Montejs Alekhandro
|
RU2711147C2 |
| GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A |
2017 |
- Wang, Lili
- Wilson, James M.
- Sidrane, Jenny Agnes
|
RU2762257C2 |
| METHODS FOR ENHANCING BIOLOGICAL ACTIVITY OF RECOMBINANT ADENO ASSOCIATED VIRUS PRODUCED BY BACULOVIRUS SYSTEM |
2017 |
- Zolotukhin, Sergej
- Kondratov, Oleksandr
|
RU2762948C2 |
| ADENO-ASSOCIATED VIRUS VECTOR |
2015 |
|
RU2743382C2 |
| GENOTHERAPY CONSTRUCTIONS FOR TREATMENT OF WILSON'S DISEASE |
2020 |
- Livingston, Christine
- Wadsworth, Samuel
|
RU2807158C2 |
| COMPOSITIONS AND METHODS OF TREATING MPS1 |
2014 |
- Uilson Dzhejms M.
- Gurda Brittni L.
|
RU2708318C2 |
| METHODS AND COMPOSITIONS FOR BRAIN DISEASES TREATMENT |
2014 |
- Devidson Beverli L.
- Tesedor Lyuis
- Chen Yun Khun
|
RU2664471C2 |
| OPTIMIZED CLN1 GENES AND EXPRESSION CASSETTES, AND THEIR APPLICATION |
2017 |
|
RU2764919C2 |
