CA3039928A1 - Aav delivery of nucleobase editors - Google Patents

Aav delivery of nucleobase editors Download PDF

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Publication number
CA3039928A1
CA3039928A1 CA3039928A CA3039928A CA3039928A1 CA 3039928 A1 CA3039928 A1 CA 3039928A1 CA 3039928 A CA3039928 A CA 3039928A CA 3039928 A CA3039928 A CA 3039928A CA 3039928 A1 CA3039928 A1 CA 3039928A1
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Canada
Prior art keywords
sggs
seq
nucleotide sequence
cas9
composition
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Pending
Application number
CA3039928A
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English (en)
French (fr)
Inventor
David R. Liu
Jonathan Ma Levy
Wei Hsi Yeh
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Harvard University
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Harvard University
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Publication of CA3039928A1 publication Critical patent/CA3039928A1/en
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/62—DNA sequences coding for fusion proteins
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04001—Cytosine deaminase (3.5.4.1)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04004—Adenosine deaminase (3.5.4.4)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
    • C07K2319/92—Fusion polypeptide containing a motif for post-translational modification containing an intein ("protein splicing")domain
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12Q2521/00—Reaction characterised by the enzymatic activity
    • C12Q2521/50—Other enzymatic activities
    • C12Q2521/539—Deaminase

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CA3039928A 2016-10-14 2017-10-13 Aav delivery of nucleobase editors Pending CA3039928A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201662408575P 2016-10-14 2016-10-14
US62/408,575 2016-10-14
US201762475780P 2017-03-23 2017-03-23
US62/475,780 2017-03-23
PCT/US2017/056671 WO2018071868A1 (en) 2016-10-14 2017-10-13 Aav delivery of nucleobase editors

Publications (1)

Publication Number Publication Date
CA3039928A1 true CA3039928A1 (en) 2018-04-19

Family

ID=60263004

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3039928A Pending CA3039928A1 (en) 2016-10-14 2017-10-13 Aav delivery of nucleobase editors

Country Status (11)

Country Link
US (2) US11306324B2 (enExample)
EP (1) EP3526320A1 (enExample)
JP (3) JP7588390B2 (enExample)
KR (2) KR20240007715A (enExample)
CN (1) CN110214180A (enExample)
AU (1) AU2017342543B2 (enExample)
CA (1) CA3039928A1 (enExample)
GB (1) GB2573062A (enExample)
IL (1) IL265900A (enExample)
SG (1) SG11201903089RA (enExample)
WO (1) WO2018071868A1 (enExample)

Cited By (1)

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Publication number Priority date Publication date Assignee Title
US12529041B2 (en) 2018-09-07 2026-01-20 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system

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EP2986635B1 (en) 2013-04-18 2018-10-03 Fondazione Telethon Effective delivery of large genes by dual aav vectors
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
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KR102796744B1 (ko) 2015-06-09 2025-04-15 에디타스 메디신, 인코포레이티드 이식의 개선을 위한 crispr/cas-관련 방법 및 조성물
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