FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology. Isolated nucleic acid is proposed to reduce the expression of the pathogenic huntingtin protein. The isolated nucleic acid contains a transgene encoding one or more mature single-stranded microRNAs, in which the nucleotide sequence of the transgene encoding each mature single-stranded microRNA contains the sequence specified under SEQ ID NO: 7, and is flanked by a heterologous spanning sequence of microRNAs. A vector for reducing the expression of the pathogenic huntingtin protein, which contains isolated nucleic acid. Recombinant AAV (rAAV) to reduce the expression of the pathogenic huntingtin protein (NTT), where the specified rAAV contains (i) capsid protein and (ii) isolated nucleic acid.
EFFECT: invention reduces the expression of the pathogenic huntingtin protein in patients with Huntington's disease.
22 cl, 55 dwg, 6 tbl, 4 ex
| Title | Year | Author | Number |
|---|---|---|---|
| RNAi AGENT OPTION | 2018 |
|
RU2789647C2 |
| THERAPEUTIC COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE | 2015 |
|
RU2711147C2 |
| VERSIONS OF RECOMBINANT AAV AND USE THEREOF | 2015 |
|
RU2738421C2 |
| ADENO-ASSOCIATED VIRAL VECTORS FOR TREATMENT OF MYOCILIN (MYOC) GLAUCOMA | 2015 |
|
RU2746991C2 |
| ADENO-ASSOCIATED VIRUS VECTORS FOR TREATING MYOCILIN (MYOC) GLAUCOMA | 2015 |
|
RU2718047C2 |
| ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MUCOLIPIDOSIS TYPE II | 2016 |
|
RU2742612C2 |
| COMPOSITIONS, METHODS AND APPLICATIONS OF GENE TRANSFER FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES | 2017 |
|
RU2805606C2 |
| GENOTHERAPY CONSTRUCTIONS FOR TREATMENT OF WILSON'S DISEASE | 2020 |
|
RU2807158C2 |
| GENE THERAPY OF OPHTHALMIC DISORDERS | 2016 |
|
RU2762747C2 |
| GENE EDITING OF DEEP INTRON MUTATIONS | 2016 |
|
RU2759335C2 |
