ADENO-ASSOCIATED VIRUS VECTORS FOR TREATING MYOCILIN (MYOC) GLAUCOMA Russian patent published in 2020 - IPC A61K48/00 A61P27/02 C12N15/864 

Abstract RU 2718047 C2

FIELD: medicine.

SUBSTANCE: group of inventions refers to medicine, namely to ophthalmology, and aims at treating myocillin (MYOC) glaucoma in a mammal. Method of treating MYOC-glaucoma involves introducing into the eye of a mammal a particle of a recombinant adeno-associated virus (rAAV) containing a vector coding RSPO1, RSPO2, RSPO3, RSPO4, truncated RSPO1, truncated RSPO2, truncated RSPO3 or truncated RSPO4. Truncated RSPO1, truncated RSPO2, truncated RSPO3 or truncated RSPO4 contains one or more furin-like domains rich in Cys, but does not have one or more of: a signal peptide, a thrombospondin domain of type 1 and/or a C-terminal positively charged domain. Another alternative to the method of treating MYOC-glaucoma in a mammal is to introduce into a mammalian eye a rAAV particle containing a vector encoding RSPO1, RSPO2, RSPO3 or RSPO4, and rAAV particles containing a vector coding agent for RNAi with respect to MYOC, wherein the RNAi agent for MYOC is a small inhibitory RNA (siRNA), a microRNA (miRNA) or a small hairpin RNA (shRNA). Above recombinant particles AAV and their containing kits for treating myocilin glaucoma in a mammal are also provided.

EFFECT: using the group of inventions makes it possible to intensify Wnt signals transmission in the mammal eye, thus providing effective treatment of myocilin glaucoma in a mammal.

20 cl, 15 dwg, 6 ex

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RU 2 718 047 C2

Authors

Pechan, Peter

Scaria, Abraham

Ardinger, Jeffery

Dates

2020-03-30Published

2015-09-16Filed