COMPOSITION AND METHOD FOR TREATING A COMPLEMENT-MEDIATED DISEASE Russian patent published in 2020 - IPC C12N15/861 C07K14/47 A61K48/00 A61K47/69 A61P7/00 A61P13/12 A61P27/02 

Abstract RU 2727411 C2

FIELD: biotechnology.

SUBSTANCE: invention relates to recombinant AAV expressing vectors, and can be used in medicine in treating a complement-related disease. Recombinant AAV vector with an expression cassette packed therein contains a nucleic acid encoding a human factor complement H (hfH) variant, functionally related to expression control sequences, which control its expression. Said version of hfH comprises a short consensus repeat (SCR) 1, 2, 3, 4, 19 and 20, and SCR 9, 10, 11, 12, 13, 14 and/or 15 are absent.

EFFECT: invention provides expressing a soluble variant of hfH protein preserving the complement regulation function.

17 cl, 37 dwg, 7 tbl

Similar patents RU2727411C2

Title Year Author Number
INTRATHECAL ADMINISTRATION OF VECTORS BASED ON ADENO ASSOCIATED VIRUSES FOR GENE THERAPY 2016
  • Wilson James M.
  • Hinderer Christian
  • Rothwell William Thomas
RU2775138C2
TREATMENT OF COMPLEMENT-MEDIATED DISORDERS 2017
  • Lachmann, Peter
  • Alexander, Ian
RU2768982C2
COMPOSITIONS AND METHODS OF TREATING MPS1 2014
  • Uilson Dzhejms M.
  • Gurda Brittni L.
RU2708318C2
COMPLEMENT FACTOR I AND COMPLEMENT FACTOR I COFACTOR, VECTORS ENCODING THEM AND USE FOR THERAPEUTIC PURPOSES 2019
  • Dreismann Anna
  • Ellis Scott
  • Joel Josephine Heather Lucienne
RU2809247C2
GENE THERAPY FOR THE TREATMENT OF HEMOPHILIA A 2017
  • Wang, Lili
  • Wilson, James M.
  • Sidrane, Jenny Agnes
RU2762257C2
ANTIBODIES AGAINST FACTOR D AND THEIR USE 2018
  • Song, Wenchao
  • Zhou, Lin
  • Sato, Sayaka
  • Miwa, Takashi
  • Gullipalli, Damodar
  • Golla, Madhu
RU2799044C2
OPTIMIZED CLN1 GENES AND EXPRESSION CASSETTES, AND THEIR APPLICATION 2017
  • Gray, Steven
RU2764919C2
COMPOSITIONS, METHODS AND APPLICATIONS OF GENE TRANSFER FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES 2017
  • Davidson, Beverly L.
  • Chen, Yong Hong
  • Tecedor, Luis
RU2805606C2
ANTI-C5a ANTIBODIES AND THEIR APPLICATIONS 2018
  • Song, Wenchao
  • Miwa, Takashi
  • Sato, Sayaka
  • Gullipalli, Damodar
RU2773779C2
METHODS FOR TREATMENT OF DANON'S DISEASE AND OTHER AUTOPHAGY DISORDERS 2017
  • Adler, Eric, D.
  • Nelson, Bradley
  • Hashem, Sherin
RU2777571C2

RU 2 727 411 C2

Authors

Song, Wenchao

Gullipalli, Damodar

Miwa, Takashi

Dates

2020-07-21Published

2016-09-23Filed