AU2014281031B2 - Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components - Google Patents

Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components Download PDF

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Publication number
AU2014281031B2
AU2014281031B2 AU2014281031A AU2014281031A AU2014281031B2 AU 2014281031 B2 AU2014281031 B2 AU 2014281031B2 AU 2014281031 A AU2014281031 A AU 2014281031A AU 2014281031 A AU2014281031 A AU 2014281031A AU 2014281031 B2 AU2014281031 B2 AU 2014281031B2
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sequence
cas9
crispr
cells
target
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AU2014281031A1 (en
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Le Cong
Matthias Heidenreich
Fei RAN
Lukasz SWIECH
Feng Zhang
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Massachusetts Institute of Technology
Broad Institute
Harvard University
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Massachusetts Institute of Technology
Broad Institute
Harvard University
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Priority claimed from PCT/US2013/074667 external-priority patent/WO2014093622A2/en
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/1034—Isolating an individual clone by screening libraries
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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AU2014281031A 2013-06-17 2014-06-11 Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for targeting disorders and diseases using viral components Active AU2014281031B2 (en)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201361836123P 2013-06-17 2013-06-17
US61/836,123 2013-06-17
US201361847537P 2013-07-17 2013-07-17
US61/847,537 2013-07-17
US201361862355P 2013-08-05 2013-08-05
US61/862,355 2013-08-05
US201361871301P 2013-08-28 2013-08-28
US61/871,301 2013-08-28
US201361915225P 2013-12-12 2013-12-12
US61/915,225 2013-12-12
PCT/US2013/074667 WO2014093622A2 (en) 2012-12-12 2013-12-12 Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
AUPCT/US2013/074667 2013-12-12
US201461979879P 2014-04-15 2014-04-15
US61/979,879 2014-04-15
PCT/US2014/041809 WO2014204729A1 (en) 2013-06-17 2014-06-11 Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components

Publications (2)

Publication Number Publication Date
AU2014281031A1 AU2014281031A1 (en) 2016-01-21
AU2014281031B2 true AU2014281031B2 (en) 2020-05-21

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EP (2) EP3597755A1 (enExample)
JP (1) JP6702858B2 (enExample)
KR (2) KR20250012194A (enExample)
CN (2) CN105793425B (enExample)
AU (1) AU2014281031B2 (enExample)
BR (1) BR122021009076B1 (enExample)
CA (1) CA2915795C (enExample)
IL (1) IL243195B (enExample)
MX (1) MX374532B (enExample)
RU (1) RU2716421C2 (enExample)
SG (2) SG10201710487VA (enExample)
WO (1) WO2014204729A1 (enExample)
ZA (1) ZA201508961B (enExample)

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BR122021009076B1 (pt) 2024-02-15
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EP3011034A1 (en) 2016-04-27
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AU2014281031A1 (en) 2016-01-21
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