FIELD: medicine.
SUBSTANCE: group of inventions relates to medicine. Disclosed is a nucleic acid that promotes the survival of photoreceptors, comprising a nucleotide sequence that encodes a human RdCVF protein, and a second nucleotide sequence that encodes an Igk signal sequence, operably linked to a nucleotide sequence encoding a human RdCVF protein, wherein the sequence encoding human RdCVF contains a recoded nucleotide sequence. Viral vector, an isolated cell containing a nucleic acid, and a pharmaceutical agent that promotes the survival of photoreceptors are disclosed. Method for preserving rod-shaped visual cells in the eye of a mammal is provided, comprising administering to a nucleic acid to a mammalian eye.
EFFECT: disclosed inventive group of inventions provides stable transfer of nucleic acid into a cell for local and/or long-term expression.
28 cl, 15 dwg, 3 tbl, 7 ex
| Title | Year | Author | Number |
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| COMPOSITIONS AND METHODS OF TREATING AND PREVENTING MACULAR DEGENERATION | 2015 |
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| VIRIONS OF AN ADENO-ASSOCIATED VIRUS WITH A VARIANT CAPSID AND METHODS FOR USING THEM | 2017 |
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| TREATMENT OF RETINITIS PIGMENTOSA | 2016 |
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| VECTOR SYSTEM BASED ON AN ADENO-ASSOCIATED VIRUS | 2017 |
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