FIELD: biotechnology.
SUBSTANCE: described is an expression vector based on an AAV virus (AAV), containing a foreign nucleic acid sequence consisting of a) a nucleotide sequence SEQ ID NO: 5 or b) a codon-modified nucleotide sequence having a homology of sequences at least 80% SEQ ID NO: 5, wherein said codon-modified nucleotide sequence encodes the same amino acid sequence as the coding area SEQ ID NO: 5. Also described is a host cell containing the AAV vector according to claim 1 and producing a protein encoded by the nucleotide sequence SEQ ID NO: 5. Proposed is a composition containing an AAV vector according to claim 1 for prevention or treatment of amyotrophic lateral sclerosis (ALS) or diabetic peripheral neuropathy (DPN).
EFFECT: application of the AAV vector according to the present invention provides a possibility of delivering the HGF hybrid gene to a subject with a high delivery yield.
4 cl, 8 dwg, 1 tbl, 7 ex
Title | Year | Author | Number |
---|---|---|---|
ADENO-ASSOCIATED VIRUS VECTOR | 2015 |
|
RU2743382C2 |
VIRIONS OF AN ADENO-ASSOCIATED VIRUS WITH A VARIANT CAPSID AND METHODS FOR USING THEM | 2017 |
|
RU2742724C1 |
GENE EDITING OF DEEP INTRON MUTATIONS | 2016 |
|
RU2759335C2 |
RNAi AGENT OPTION | 2018 |
|
RU2789647C2 |
THERAPEUTIC COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE | 2015 |
|
RU2711147C2 |
COMPOSITION FOR PREVENTION OR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS USING TWO OR MORE HEPATOCYTES GROWTH FACTOR ISOPHORMS | 2014 |
|
RU2639582C2 |
TREATMENT OF NEUROPATHY WITH DNA STRUCTURES ENCODING IGF-1 AND DNA STRUCTURES ENCODING HGF | 2019 |
|
RU2781980C2 |
VIRIONS OF ADENO-ASSOCIATED VIRUS WITH OPTIONAL CAPSID AND METHODS OF THEIR USE | 2012 |
|
RU2611202C2 |
RECOMBINANT VIRAL VECTORS WITH MODIFIED TROPISM AND WAYS OF THEIR USE FOR TARGETED INTRODUCTION OF GENETIC MATERIAL INTO HUMAN CELLS | 2018 |
|
RU2809246C2 |
INTRATHECAL ADMINISTRATION OF VECTORS BASED ON ADENO ASSOCIATED VIRUSES FOR GENE THERAPY | 2016 |
|
RU2775138C2 |
Authors
Dates
2021-12-20—Published
2018-12-28—Filed