FIELD: biotechnology.
SUBSTANCE: invention relates to the field of biotechnology. A drug for the treatment of metachromatic leukodystrophy is described, including a recombinant adeno-associated serotype 9 virus containing a codon-optimized sequence of the ARSA gene represented by SEQ ID NO: 1, a drug for the treatment of metachromatic leukodystrophy consisting of mesenchymal stem cells genetically modified with a recombinant adeno-associated serotype 9 virus containing a codon-optimized sequence of the ARSA gene represented in SEQ ID NO: 1, and a method for treating metachromatic leukodystrophy. In one embodiment, the method for treatment of metachromatic leukodystrophy includes a single intravenous injection of the above drugs or a single intrathecal injection of a drug including a recombinant adeno-associated virus of serotype 9.
EFFECT: invention expands the arsenal of tools for gene and gene-cell therapy of metachromatic leukodystrophy.
3 cl, 6 dwg, 1 tbl, 3 ex
| Title | Year | Author | Number |
|---|---|---|---|
| RECOMBINANT GENETIC CONSTRUCT, ADENO-ASSOCIATED VIRUS FOR THERAPY OF METACHROMATIC LEUKODYSTROPHY | 2023 |
|
RU2821568C1 |
| METHOD FOR THERAPY FOR TAY-SACHS DISEASE AND SANDHOFF DISEASE USING GENETICALLY MODIFIED HUMAN MESENCHYMAL STEM CELLS WITH OVEREXPRESSION OF β-HEXOSAMINIDASE A | 2020 |
|
RU2748383C1 |
| METHODS AND COMPOSITIONS FOR DELIVERY OF ARYLSULFATASE A TO CNS | 2011 |
|
RU2783380C2 |
| VECTORS AND SEQUENCES FOR TREATING DISEASES | 2011 |
|
RU2588667C2 |
| COMPOSITIONS, METHODS AND APPLICATIONS OF GENE TRANSFER FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES | 2017 |
|
RU2805606C2 |
| METHODS AND COMPOSITIONS FOR CNS DELIVERY OF ARYLSULFATASE A | 2011 |
|
RU2671503C2 |
| METHODS AND COMPOSITIONS FOR BRAIN DISEASES TREATMENT | 2014 |
|
RU2664471C2 |
| VECTORS FOR TREATMENT OF FRIEDREICH'S ATAXIA | 2018 |
|
RU2774892C2 |
| VECTORS BASED ON ADENO-ASSOCIATED VIRUSES FOR TREATING MUCOPOLYSACCHARIDOSES | 2016 |
|
RU2744593C2 |
| INTRATHECAL ADMINISTRATION OF VECTORS BASED ON ADENO ASSOCIATED VIRUSES FOR GENE THERAPY | 2016 |
|
RU2775138C2 |
