FIELD: biotechnology.
SUBSTANCE: invention relates to a nucleic acid construct containing a 5'-inverted terminal repeat (ITR) sequence, a promoter sequence, a nucleic acid sequence encoding truncated human copper-transporting ATPase 2 (ATP7B), a 3'-ITR sequence, and also to the composition containing it.
EFFECT: invention is effective for the treatment of Wilson's disease.
20 cl, 12 dwg, 6 ex
| Title | Year | Author | Number |
|---|---|---|---|
| NUCLEIC ACID CONSTRUCTIONS AND VECTORS FOR GENOTHERAPY FOR TREATING WILSON'S DISEASE AND OTHER CONDITIONS | 2015 |
|
RU2745567C2 |
| RECOMBINANT AAV VECTORS EXPRESSING OSTEOPROTECTIVE GENES, INCLUDING HAS2 AND LUBRICIN, SUITABLE IN TREATMENT OF OSTEOARTHRITIS AND SIMILAR JOINT DISEASES IN MAMMALS | 2017 |
|
RU2771490C2 |
| GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A | 2017 |
|
RU2762257C2 |
| NEW LIVER TARGETING ADENO-ASSOCIATED VIRAL VECTORS | 2019 |
|
RU2793735C2 |
| ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MUCOLIPIDOSIS TYPE II | 2016 |
|
RU2742612C2 |
| TREATMENT OF COMPLEMENT-MEDIATED DISORDERS | 2017 |
|
RU2768982C2 |
| METHODS FOR AAV DETECTION | 2017 |
|
RU2771622C2 |
| RECOVERED NUCLEIC ACID WHICH CODES FVIII-BDD-BASED FUSION PROTEIN AND HETEROLOGOUS SIGNAL PEPTIDE, AND USE THEREOF | 2022 |
|
RU2818229C2 |
| OPTIMIZED GENES AND EXPRESSION CASSETTES OF MINI-DYSTROPHIN AND THEIR APPLICATION | 2017 |
|
RU2753194C2 |
| ADENO-ASSOCIATED VIRUS VECTORS FOR TREATING MYOCILIN (MYOC) GLAUCOMA | 2015 |
|
RU2718047C2 |
