FIELD: biotechnology.
SUBSTANCE: group of inventions relates to agents for use in gene therapy. Disclosed is a polynucleotide coding factor H-like protein 1 (FHL1), a vector containing said polynucleotide, a cell for expressing FHL1 and their use for the treatment or prevention of complement-mediated disorder associated with complement feedback cycle hyperactivity, including eye diseases such as age-related macular degeneration (AMD) or diabetic retinopathy. Nucleotide sequence coding FHL1 is at least 90% identical to the sequence SEQ ID NO: 12.
EFFECT: inventions make it possible to deliver to the patient higher doses of the corresponding protein without increasing the amount of the introduced vector.
37 cl, 6 dwg, 2 tbl, 1 ex
| Title | Year | Author | Number |
|---|---|---|---|
| COMPLEMENT FACTOR I AND COMPLEMENT FACTOR I COFACTOR, VECTORS ENCODING THEM AND USE FOR THERAPEUTIC PURPOSES | 2019 |
|
RU2809247C2 |
| GENE THERAPY | 2016 |
|
RU2740038C2 |
| TREATMENT OF COMPLEMENT-MEDIATED DISORDERS | 2017 |
|
RU2768982C2 |
| TREATMENT OF RETINITIS PIGMENTOSA | 2016 |
|
RU2737049C2 |
| ADENO-ASSOCIATED VIRUS VECTOR | 2015 |
|
RU2743382C2 |
| VIRIONS OF AN ADENO-ASSOCIATED VIRUS WITH A VARIANT CAPSID AND METHODS FOR USING THEM | 2017 |
|
RU2742724C1 |
| COMPOSITION AND METHOD FOR TREATING A COMPLEMENT-MEDIATED DISEASE | 2016 |
|
RU2727411C2 |
| VIRIONS OF ADENO-ASSOCIATED VIRUS WITH OPTIONAL CAPSID AND METHODS OF THEIR USE | 2012 |
|
RU2611202C2 |
| METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISORDERS AND DISEASES RELATED TO RDH12 | 2017 |
|
RU2764920C2 |
| COMPOSITIONS AND METHODS OF TREATING AND PREVENTING MACULAR DEGENERATION | 2015 |
|
RU2703145C2 |
