FIELD: biotechnology.
SUBSTANCE: invention relates to biotechnology. Described is a recombinant adeno-associated virus (rAAV) vector comprising nucleic acid encoding alpha and beta subunits N-acetylglucosamine-1 -phosphate transferase, (GNPTAB) and at least one AAV inverted terminal repeat (ITR).
EFFECT: invention expands the range of methods for treating mucolipidosis type II (ML II) or mucolipidosis type III (ML III) in a mammal or relating to increasing body size, bone mineral content and/or bone mineral density in a mammal with mucolipidosis type II (ML II) or mucolipidosis type III (ML III).
22 cl, 12 dwg, 4 tbl, 2 ex
| Title |
Year |
Author |
Number |
| GENOTHERAPY CONSTRUCTIONS FOR TREATMENT OF WILSON'S DISEASE |
2020 |
- Livingston, Christine
- Wadsworth, Samuel
|
RU2807158C2 |
| METHODS FOR AAV DETECTION |
2017 |
- Jin, Xiaoying
- O'Riordan, Catherine, R.
- Liu, Lin
- Zhang, Kate
|
RU2771622C2 |
| TREATMENT OF COMPLEMENT-MEDIATED DISORDERS |
2017 |
- Lachmann, Peter
- Alexander, Ian
|
RU2768982C2 |
| NUCLEIC ACID CONSTRUCTIONS AND VECTORS FOR GENOTHERAPY FOR TREATING WILSON'S DISEASE AND OTHER CONDITIONS |
2015 |
- Murillo Sauca, Oihana
- Gonzalez Aseguinolaza, Gloria
- Hernandez Alcoceba, Ruben
|
RU2745567C2 |
| ADENO-ASSOCIATED VIRUS VECTORS FOR TREATING MYOCILIN (MYOC) GLAUCOMA |
2015 |
- Pechan, Peter
- Scaria, Abraham
- Ardinger, Jeffery
|
RU2718047C2 |
| RECOMBINANT AAV VECTORS EXPRESSING OSTEOPROTECTIVE GENES, INCLUDING HAS2 AND LUBRICIN, SUITABLE IN TREATMENT OF OSTEOARTHRITIS AND SIMILAR JOINT DISEASES IN MAMMALS |
2017 |
- Dias Figejredo Monika
- Kuostio-Mor Sirka Rm
- Bertelet Patrisiya
|
RU2771490C2 |
| NEW LIVER TARGETING ADENO-ASSOCIATED VIRAL VECTORS |
2019 |
- Colosi, Peter Cameron
- Ramirez, Silvia
|
RU2793735C2 |
| MODIFIED FACTOR IX, AS WELL AS COMPOSITIONS, METHODS AND OPTIONS FOR THE USE OF GENE TRANSFER INTO CELLS, ORGANS AND TISSUE |
2016 |
- Khaj Ketrin A.
- Angela Khaver
|
RU2811445C2 |
| GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A |
2017 |
- Wang, Lili
- Wilson, James M.
- Sidrane, Jenny Agnes
|
RU2762257C2 |
| OPTIMIZED GENES AND EXPRESSION CASSETTES OF MINI-DYSTROPHIN AND THEIR APPLICATION |
2017 |
- Mcphee, Scott W.J.
- Qiao, Chunping
- Li, Juan
- Xiao, Xiao
- Mcintyre, Maritza
- Samulski, Richard J.
|
RU2753194C2 |
