CA2986021A1 - Gene editing of deep intronic mutations - Google Patents

Gene editing of deep intronic mutations Download PDF

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Publication number
CA2986021A1
CA2986021A1 CA2986021A CA2986021A CA2986021A1 CA 2986021 A1 CA2986021 A1 CA 2986021A1 CA 2986021 A CA2986021 A CA 2986021A CA 2986021 A CA2986021 A CA 2986021A CA 2986021 A1 CA2986021 A1 CA 2986021A1
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Prior art keywords
guide rna
cas
promoter
composition
seq
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CA2986021A
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English (en)
French (fr)
Inventor
Guoxiang RUAN
Abraham Scaria
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Genzyme Corp
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Genzyme Corp
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Publication of CA2986021A1 publication Critical patent/CA2986021A1/en
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CA2986021A 2015-05-16 2016-04-15 Gene editing of deep intronic mutations Pending CA2986021A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562162720P 2015-05-16 2015-05-16
US62/162,720 2015-05-16
PCT/US2016/027987 WO2016186772A2 (en) 2015-05-16 2016-04-15 Gene editing of deep intronic mutations

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CA2986021A1 true CA2986021A1 (en) 2016-11-24

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US (2) US11896651B2 (enExample)
EP (1) EP3298134B8 (enExample)
JP (3) JP6985250B2 (enExample)
KR (2) KR20230142649A (enExample)
CN (3) CN118879692A (enExample)
AU (2) AU2016265255B2 (enExample)
BR (1) BR112017024514A2 (enExample)
CA (1) CA2986021A1 (enExample)
ES (1) ES2952598T3 (enExample)
IL (2) IL296365A (enExample)
MX (2) MX2017014665A (enExample)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019183641A1 (en) * 2018-03-23 2019-09-26 Massachusetts Eye And Ear Infirmary Crispr/cas9-mediated exon-skipping approach for ush2a-associated usher syndrome

Families Citing this family (75)

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EP2734621B1 (en) 2011-07-22 2019-09-04 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
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
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
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