Processing

Please wait...

Settings

Settings

Goto Application

1. WO2021030666 - BASE EDITING BY TRANSGLYCOSYLATION

Publication Number WO/2021/030666
Publication Date 18.02.2021
International Application No. PCT/US2020/046320
International Filing Date 14.08.2020
IPC
C12N 9/10 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
10Transferases (2.)
C12N 9/22 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
CPC
C12N 9/1022
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes; Proenzymes; Compositions thereof
10Transferases (2.)
1022transferring aldehyde or ketonic groups (2.2)
C12N 9/22
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes; Proenzymes; Compositions thereof
14Hydrolases (3)
16acting on ester bonds (3.1)
22Ribonucleases ; RNAses, DNAses
Applicants
  • THE BROAD INSTITUTE, INC. [US]/[US]
  • PRESIDENT AND FELLOWS OF HARVARD COLLEGE [US]/[US]
Inventors
  • LIU, David, R.
  • MERCER, Jaron August McClure
Agents
  • GREENWALD, Eric P.
  • BAKER, C., Hunter
  • ATTISHA, Michael, J.
  • ALAM, Saad
  • AMUNDSEN, Eric, L.
Priority Data
62/887,30715.08.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) BASE EDITING BY TRANSGLYCOSYLATION
(FR) ÉDITION DE BASES PAR TRANSGLYCOSYLATION
Abstract
(EN)
The present specification provides for base editors that satisfy a need in the art for installation of targeted transversions of thymine (T) to guanine (G), or correspondingly, transversions of adenine (A) to cytosine (C). The base editors include a nucleic acid programmable DNA binding protein domain and a transglycosylase domain. The base editors may be engineered through the use of continuous or non-continuous evolution systems. The present specification provides for thymine-to-guanine (or adenine-to-cytosine) base editor variants that can install single-base transversion mutations in DNA. In addition, methods for targeted nucleic acid editing are provided. Further provided are pharmaceutical compositions comprising the disclosed base editors, and vectors and kits useful for the generation of these base editors. In some embodiments, cells containing such vectors are provided. Further provided are methods comprising contacting a cell with the base editors and methods comprising administering the base editors to a subject.
(FR)
La présente invention concerne des éditeurs de base satisfaisant un besoin dans l'état de la technique pour l'installation de transversions ciblées de thymine (T) en guanine (G), ou de manière correspondante, des transversions d'adénine (A) en cytosine (C). Les éditeurs de base comprennent un domaine de protéine de liaison à l'ADN programmable par un acide nucléique et un domaine de transglycosylase. Les éditeurs de bases peuvent être modifiés au moyen de systèmes d'évolution continu ou non continu. La présente invention concerne des variants d'éditeur de base thymine-à-guanine (ou adénine-à-cytosine) pouvant installer des mutations de transversion à base unique dans l'ADN. En outre, l'invention porte sur des procédés d'édition ciblée d'acides nucléiques. L'invention concerne en outre des compositions pharmaceutiques comprenant les éditeurs de bases de l'invention, et des vecteurs et des kits utiles pour la génération de ces éditeurs de bases. Selon certains autres modes de réalisation, l'invention concerne des cellules contenant ces vecteurs. L'invention concerne en outre des procédés comprenant la mise en contact d'une cellule avec les éditeurs de bases et des procédés comprenant l'administration des éditeurs de bases à un sujet.
Latest bibliographic data on file with the International Bureau