FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology, specifically to adeno-associated virus (AAV) vectors for delivery of a heterologous polynucleotide sequence coding a therapeutic protein, and can be used in medicine for treating lysosomal storage disease. Recombinant AAV (rAAV) particle is constructed containing the AAV capsid sequence SEQ ID NO: 5. RAAV gene contains an inverted terminal repeat AAV (ITR) and a heterologous polynucleotide sequence which codes an enzyme involved in the development of lysosomal storage disease and which is functionally linked to an expression control element and is flanked by 5' and/or 3' AAV ITR.
EFFECT: invention provides efficient delivery of a heterologous polynucleotide sequence coding an enzyme involved in developing lysosomal storage disease.
35 cl, 6 dwg, 4 tbl, 9 ex
| Title | Year | Author | Number |
|---|---|---|---|
| MODIFIED FACTOR IX, AS WELL AS COMPOSITIONS, METHODS AND OPTIONS FOR THE USE OF GENE TRANSFER INTO CELLS, ORGANS AND TISSUE | 2016 |
|
RU2811445C2 |
| COMPOSITIONS AND METHODS FOR INCREASING OR ENHANCING TRANSDUCTION OF GENE THERAPY VECTORS AND FOR REMOVING OR REDUCING IMMUNOGLOBULINS | 2021 |
|
RU2841237C1 |
| VARIANTS OF ACID ALPHA-GLUCOSIDASE AND THEIR USE | 2017 |
|
RU2780410C2 |
| GENE THERAPY OF HYPOPHOSPHATEMIC DISEASES ASSOCIATED WITH FIBROBLAST GROWTH FACTOR 23 | 2020 |
|
RU2815545C2 |
| METHODS OF GENOTHERAPY USING FACTOR VIII (FVIII) GENE | 2018 |
|
RU2808274C2 |
| CPG REDUCED FACTOR VIII VARIANTS, COMPOSITIONS AND METHODS AND USE FOR TREATMENT OF HEMOSTASIS DISORDERS | 2016 |
|
RU2745506C2 |
| OPTIONS OF ACID ALPHA-GLUCOSIDASE AND THEIR USE | 2017 |
|
RU2780329C2 |
| NEW LIVER TARGETING ADENO-ASSOCIATED VIRAL VECTORS | 2019 |
|
RU2793735C2 |
| CODON-OPTIMIZED NUCLEIC ACID WHICH ENCODES SMN1 PROTEIN, AND USE THEREOF | 2020 |
|
RU2742837C1 |
| NON-DESTRUCTIVE GENE THERAPY FOR TREATING MMA | 2018 |
|
RU2820602C2 |
