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1. WO2021243174 - DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF SAMD9L

Publication Number WO/2021/243174
Publication Date 02.12.2021
International Application No. PCT/US2021/034794
International Filing Date 28.05.2021
IPC
C12Q 1/6881 2018.1
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
6881for tissue or cell typing, e.g. human leukocyte antigen probes
C12Q 1/6827 2018.1
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12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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1Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
68involving nucleic acids
6813Hybridisation assays
6827for detection of mutation or polymorphism
C12N 9/22 2006.1
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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 15/111
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
111General methods applicable to biologically active non-coding nucleic acids
C12N 15/1135
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15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
1135against oncogenes or tumor suppressor genes
C12N 2310/20
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2310Structure or type of the nucleic acid
10Type of nucleic acid
20involving clustered regularly interspaced short palindromic repeats [CRISPRs]
C12N 2320/34
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2320Applications; Uses
30Special therapeutic applications
34Allele or polymorphism specific uses
Applicants
  • EMENDOBIO INC. [US]/[US] (AllExceptUS)
  • EMMANUEL, Rafi [IL]/[IL] (US)
  • DICKEN, Joseph [IL]/[IL] (US)
  • GOLAN MASHIACH, Michal [IL]/[IL] (US)
Inventors
  • EMMANUEL, Rafi
  • DICKEN, Joseph
  • GOLAN MASHIACH, Michal
Agents
  • GERSHIK, Gary J.
Priority Data
63/031,15728.05.2020US
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF SAMD9L
(FR) INACTIVATION DIFFÉRENTIELLE D'UN ALLÈLE HÉTÉROZYGOTE DE SAMD9L
Abstract
(EN) RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-20246 and compositions, methods, and uses thereof.
(FR) L'invention concerne des molécules d'ARN comprenant une partie de séquence guide ayant 17-50 nucléotides contigus contenant des nucléotides dans la séquence présentée dans l'une quelconque des SEQ ID NO : 1-20646, ainsi que des compositions, des procédés et des utilisations associées.
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