FIELD: biotechnology.
SUBSTANCE: group of inventions relates to biotechnology, in particular to the use of an isolated nucleic acid molecule for the expression of an exogenous gene in a cell of ape retinal pigment epithelium and for the treatment of a disease associated with retinal pigment epithelium. The isolated nucleic acid molecule containing or consisting of a nucleic acid sequence under SEQ ID NO: 1 is delivered to cells of ape retinal pigment epithelium, wherein the specified isolated nucleic acid molecule is functionally connected to the nucleic acid sequence encoding the exogenous gene.
EFFECT: invention provides specific expression of an exogenous gene in cells of ape retinal pigment epithelium, and it can be used to direct and target gene expression into cells of retinal pigment epithelium in retinal lesions, for example, in patients with age-related macular degeneration, retinal pigment dystrophy, diabetic retinopathy or retinal pigment epithelium hypertrophy.
16 cl, 1 dwg, 1 ex
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|
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| DISEASE-RELATED PROTEIN | 2002 |
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|
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| POLYPEPTIDES DERIVED FROM TRYPTOPHANYL-TRNA-SYNTHETASE AND USES THEREOF IN CONTROLLING OF VASCULARISATION | 2002 |
|
RU2297425C2 |
| B4GALT1 OPTIONS AND THEIR APPLICATIONS | 2018 |
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| FAD2 PERFORMANCE LOCI AND CORRESPONDING TARGET SITE SPECIFIC BINDING PROTEINS CAPABLE OF INDUCING TARGETED BREAKS | 2013 |
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| ADENO-ASSOCIATED VIRUS VECTOR | 2015 |
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