SG10201902574RA - Adeno-associated virus vector variants for high efficiency genome editing and methods thereof - Google Patents

Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

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Publication number
SG10201902574RA
SG10201902574RA SG10201902574RA SG10201902574RA SG10201902574RA SG 10201902574R A SG10201902574R A SG 10201902574RA SG 10201902574R A SG10201902574R A SG 10201902574RA SG 10201902574R A SG10201902574R A SG 10201902574RA SG 10201902574R A SG10201902574R A SG 10201902574RA
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Singapore
Prior art keywords
methods
vector
subject
adeno
associated virus
Prior art date
Application number
SG10201902574RA
Inventor
Saswati Chatterjee
Laura Smith
Kamehameha Wong
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Hope City
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Publication date
Application filed by Hope City filed Critical Hope City
Publication of SG10201902574RA publication Critical patent/SG10201902574RA/en

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    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/24—Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

Abstract

ADENO-ASSOCIATED VIRUS VECTOR VARIANTS FOR IDGH EFFICIENCY GENOME EDITING AND METHODS THEREOF Adeno-associated virus (AA V) Clade F vectors or AA V vector variants (relative to AA V) for precise editing of the genome of a cell and methods and kits thereof are provided. Targeted genome editing using the AA V Clade F vectors or AA V vector variants provided herein occurred at frequencies that were shown to be 1,000 to 100,000 fold more efficient than has previously been reported. Also provided are methods of treating a disease or disorder in a subject by editing the genome of a cell of the subject via transducing the cell with an AA V Clade F vector or AA V vector variant as described herein and further transplanting the transduced cell into the subject to treat the disease or disorder of the subject. Also provided herein are methods of treating a disease or disorder in a subject by in vivo genome editing by directly administering the AA V Clade F vector or AA V vector variant as described herein to the subject. Figure for publication: None 122
SG10201902574RA 2014-09-24 2015-09-23 Adeno-associated virus vector variants for high efficiency genome editing and methods thereof SG10201902574RA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462054899P 2014-09-24 2014-09-24
US201462063587P 2014-10-14 2014-10-14
US201562209862P 2015-08-25 2015-08-25

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US (4) US9890396B2 (en)
EP (2) EP3800260A1 (en)
JP (3) JP6683691B2 (en)
KR (2) KR102526711B1 (en)
CN (2) CN113667696A (en)
AU (2) AU2015320694B2 (en)
BR (1) BR112017005892A2 (en)
CA (1) CA2961555A1 (en)
DK (1) DK3198018T3 (en)
ES (1) ES2856090T3 (en)
MX (2) MX2017003815A (en)
PL (1) PL3198018T3 (en)
PT (1) PT3198018T (en)
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