FIELD: biotechnology.
SUBSTANCE: described is a factor VIII (FVIII) expression vector based on an adeno-associated virus (AAV). The vector comprises a liver-specific transcription regulation area and a codon-optimised area encoding a functionally active FVIII. Said area encoding a functionally active FVIII comprises nucleotides 923-5296 SEQ ID NO: 9. The vector has a length of less than 5.0 kbp.
EFFECT: invention expands the range of means for treating hemophilia A.
16 cl, 10 dwg, 6 ex
| Title |
Year |
Author |
Number |
| VECTORS OF FVIII FACTOR BASED ON ADENO-ASSOCIATED VIRUSES, CORRESPONDING VIRUS PARTICLES AND THERAPEUTIC COMPOSITIONS CONTAINING THEM |
2016 |
- Bunting, Stuart
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- Pungor, Erno
|
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2014 |
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|
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2019 |
|
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2020 |
- Kerr, Duglas Antoni
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- Silver, Nataniel
- Chiokko, Mettyu
|
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| CLOSED-ENDED DNA VECTORS OBTAINED BY CELL-FREE SYNTHESIS, AND METHOD OF PRODUCING CEDNA VECTORS |
2019 |
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|
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2020 |
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|
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2019 |
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|
RU2800914C2 |
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2019 |
- Kerr, Duglas A.
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|
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2020 |
- Livingston, Christine
- Wadsworth, Samuel
|
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| OPTIMIZED GENES AND EXPRESSION CASSETTES OF MINI-DYSTROPHIN AND THEIR APPLICATION |
2017 |
- Mcphee, Scott W.J.
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- Li, Juan
- Xiao, Xiao
- Mcintyre, Maritza
- Samulski, Richard J.
|
RU2753194C2 |
