SG10201804974RA - Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders - Google Patents

Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders

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Publication number
SG10201804974RA
SG10201804974RA SG10201804974RA SG10201804974RA SG10201804974RA SG 10201804974R A SG10201804974R A SG 10201804974RA SG 10201804974R A SG10201804974R A SG 10201804974RA SG 10201804974R A SG10201804974R A SG 10201804974RA SG 10201804974R A SG10201804974R A SG 10201804974RA
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Singapore
Prior art keywords
methods
compositions
systems
nucleotide repeat
crispr
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SG10201804974RA
Inventor
Zhang Feng
Davidson Beverly
Lin Chie-Yu
Rodriguez Edgardo
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Broad Inst Inc
Massachusetts Inst Technology
Univ Iowa Res Found
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Publication date
Application filed by Broad Inst Inc, Massachusetts Inst Technology, Univ Iowa Res Found filed Critical Broad Inst Inc
Publication of SG10201804974RA publication Critical patent/SG10201804974RA/en

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Abstract

COMPOSITIONS AND MET HODS OF USE OF CRISP R - CAS SYSTEMS IN NUCLEOTIDE REPEAT DI SORDERS The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target 5 sequences. Provided are delivery systems and tissues or organ which are targeted as sites for deliver y . Also provided are vectors and vector systems some of which encode one or more co mponents of a SIN CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing SIN CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxi city 10 and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition. Figure 1 15
SG10201804974RA 2013-12-12 2014-12-12 Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders SG10201804974RA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361915150P 2013-12-12 2013-12-12
US201462010888P 2014-06-11 2014-06-11
US201462010879P 2014-06-11 2014-06-11

Publications (1)

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SG10201804974RA true SG10201804974RA (en) 2018-07-30

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SG10201804974RA SG10201804974RA (en) 2013-12-12 2014-12-12 Compositions and Methods of Use of Crispr-Cas Systems in Nucleotide Repeat Disorders

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US (5) US20160354487A1 (en)
EP (4) EP3080258A1 (en)
JP (2) JP6625055B2 (en)
KR (2) KR20160097338A (en)
CN (2) CN106029880A (en)
AU (2) AU2014362248A1 (en)
BR (2) BR112016013520A2 (en)
CA (2) CA2932472A1 (en)
IL (2) IL246118A0 (en)
MX (2) MX2016007325A (en)
SG (2) SG10201804973TA (en)
WO (2) WO2015089351A1 (en)

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