BR112017024514A2 - edição genética de mutações intrônicas profundas - Google Patents

edição genética de mutações intrônicas profundas

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Publication number
BR112017024514A2
BR112017024514A2 BR112017024514-0A BR112017024514A BR112017024514A2 BR 112017024514 A2 BR112017024514 A2 BR 112017024514A2 BR 112017024514 A BR112017024514 A BR 112017024514A BR 112017024514 A2 BR112017024514 A2 BR 112017024514A2
Authority
BR
Brazil
Prior art keywords
genetic editing
deep intronic
intronic mutations
crispr
cas
Prior art date
Application number
BR112017024514-0A
Other languages
English (en)
Portuguese (pt)
Inventor
Ruan Guoxiang
Scaria Abraham
Original Assignee
Genzyme Corporation
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of BR112017024514A2 publication Critical patent/BR112017024514A2/pt

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    • C12N15/113—Non-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
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BR112017024514-0A 2015-05-16 2016-04-15 edição genética de mutações intrônicas profundas BR112017024514A2 (pt)

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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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EP3613852A3 (en) 2011-07-22 2020-04-22 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
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