VARIANT AAV, COMPOSITIONS AND METHODS, IN WHICH IT IS USED, AS WELL AS METHODS OF ITS APPLICATION FOR TRANSFER OF GENES INTO CELLS, ORGANS AND TISSUES Russian patent published in 2019 - IPC C07K14/15 C07K14/745 C12N7/04 C12N15/861 A61K38/36 A61K48/00 A61P7/04 

Abstract RU 2697444 C2

FIELD: biotechnology.

SUBSTANCE: invention relates to biotechnology, specifically to vectors based on the serotype AAV-Rh74 adenoassociated virus (AAV) containing a polynucleotide sequence coding human factor IX and can be used in medicine.

EFFECT: obtained particles are used in a pharmaceutical composition for effective treatment of hemophilia B.

21 cl, 7 dwg, 4 tbl, 9 ex

Similar patents RU2697444C2

Title Year Author Number
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
AAV VECTOR COMPOSITIONS AND METHODS FOR GENE TRANSFER TO CELLS, ORGANS AND TISSUES 2013
  • Khaj Katerine A.
  • Mingozzi Federiko
  • Sun Yunvej
  • Dzhonson Filip
RU2653444C2
VECTORS CONTAINING SPACER/FILLER POLYNUCLEOTIDE SEQUENCES, AND METHODS OF USING THEM 2014
  • Rajt Dzh. Frejzer
  • Zelenaya Olga
RU2725813C2
AAV VECTOR AND NEUTRALIZING ANTIBODIES AGAINST AAV (ADENO-ASSOCIATED VIRUS) ANALYSIS 2014
  • Khaj Ketrin A.
  • Mingozzi Federiko
  • Chen Ifyn
RU2705249C2
MODIFIED FREEDREICH ATAXY GENES AND VECTORS FOR GENE THERAPY 2016
  • Samulski, Richard J.
RU2743792C2
COMPOSITION AND METHODS FOR HIGHLY EFFICIENT GENE TRANSFER USING AAV CAPSID VARIANTS 2013
  • Yazisioglu Mustafa N.
  • Mingozzi Federiko
  • Angela Khaver
  • Khaj Ketrin A.
RU2683497C2
ENHANCEMENT OF TISSUE-SPECIFIC GENE DELIVERY BY CAPSID MODIFICATION 2019
  • Loiler, Scott Allen
RU2801217C2
COMPOSITIONS, METHODS AND APPLICATIONS OF GENE TRANSFER FOR THE TREATMENT OF NEURODEGENERATIVE DISEASES 2017
  • Davidson, Beverly L.
  • Chen, Yong Hong
  • Tecedor, Luis
RU2805606C2
VIRIONS OF AN ADENO-ASSOCIATED VIRUS WITH A VARIANT CAPSID AND METHODS FOR USING THEM 2017
  • Sheffer Devid V.
  • Klimchak Rajan R.
  • Kerber Dzhejms T.
  • Flenneri Dzhon Dzh.
  • Dalkara Mourot Deniz
  • Vizel Majke
  • Bern Lea S.T.
RU2742724C1
RECOMBINANT ADEN-ASSOCIATED VIRUS, WHICH IS HYBRID OF AAV9 AND AAVrh74 SEROTYPES, WITH REDUCED TROPISM FOR LIVER TISSUE 2019
  • Rishar, Izabel
  • Zhikkel, Evelin
  • Mingotstsi, Federiko
  • Rontsitti, Dzhuzeppe
  • Vidal, Patris
RU2812279C2

RU 2 697 444 C2

Authors

Khaj Ketrin A.

Yazisioglu Mustafa N.

Angela Khaver

Dates

2019-08-14Published

2014-07-22Filed