Correction of a genetic disease in mouse via use of CRISPR-Cas9

The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts...

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Vydáno v:	Cell stem cell Ročník 13; číslo 6; s. 659
Hlavní autoři:	Wu, Yuxuan, Liang, Dan, Wang, Yinghua, Bai, Meizhu, Tang, Wei, Bao, Shiming, Yan, Zhiqiang, Li, Dangsheng, Li, Jinsong
Médium:	Journal Article
Jazyk:	angličtina
Vydáno:	United States 05.12.2013
Témata:	Animals Base Sequence Cataract - genetics Cataract - therapy Clustered Regularly Interspaced Short Palindromic Repeats - genetics CRISPR-Associated Proteins - metabolism Disease Models, Animal gamma-Crystallins - genetics gamma-Crystallins - therapeutic use Genetic Therapy Mice Molecular Sequence Data RNA, Messenger - genetics RNA, Messenger - metabolism
ISSN:	1875-9777, 1875-9777
On-line přístup:	Zjistit podrobnosti o přístupu
Tagy:	Přidat tag Žádné tagy, Buďte první, kdo vytvoří štítek k tomuto záznamu!

Abstract	The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant allele. Correction occurred via homology-directed repair (HDR) based on an exogenously supplied oligonucleotide or the endogenous WT allele, with only rare evidence of off-target modifications. The resulting mice were fertile and able to transmit the corrected allele to their progeny. Thus, our study provides proof of principle for use of the CRISPR-Cas9 system to correct genetic disease.
AbstractList	The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant allele. Correction occurred via homology-directed repair (HDR) based on an exogenously supplied oligonucleotide or the endogenous WT allele, with only rare evidence of off-target modifications. The resulting mice were fertile and able to transmit the corrected allele to their progeny. Thus, our study provides proof of principle for use of the CRISPR-Cas9 system to correct genetic disease.The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant allele. Correction occurred via homology-directed repair (HDR) based on an exogenously supplied oligonucleotide or the endogenous WT allele, with only rare evidence of off-target modifications. The resulting mice were fertile and able to transmit the corrected allele to their progeny. Thus, our study provides proof of principle for use of the CRISPR-Cas9 system to correct genetic disease. The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of this system for efficient correction of a genetic disease. Here we show that mice with a dominant mutation in Crygc gene that causes cataracts could be rescued by coinjection into zygotes of Cas9 mRNA and a single-guide RNA (sgRNA) targeting the mutant allele. Correction occurred via homology-directed repair (HDR) based on an exogenously supplied oligonucleotide or the endogenous WT allele, with only rare evidence of off-target modifications. The resulting mice were fertile and able to transmit the corrected allele to their progeny. Thus, our study provides proof of principle for use of the CRISPR-Cas9 system to correct genetic disease.
Author	Wang, Yinghua Tang, Wei Yan, Zhiqiang Wu, Yuxuan Li, Dangsheng Bai, Meizhu Bao, Shiming Li, Jinsong Liang, Dan
Author_xml	– sequence: 1 givenname: Yuxuan surname: Wu fullname: Wu, Yuxuan organization: Group of Epigenetic Reprogramming, State Key Laboratory of Cell Biology, Shanghai Key Laboratory of Molecular Andrology, Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, Shanghai, 200031, China – sequence: 2 givenname: Dan surname: Liang fullname: Liang, Dan – sequence: 3 givenname: Yinghua surname: Wang fullname: Wang, Yinghua – sequence: 4 givenname: Meizhu surname: Bai fullname: Bai, Meizhu – sequence: 5 givenname: Wei surname: Tang fullname: Tang, Wei – sequence: 6 givenname: Shiming surname: Bao fullname: Bao, Shiming – sequence: 7 givenname: Zhiqiang surname: Yan fullname: Yan, Zhiqiang – sequence: 8 givenname: Dangsheng surname: Li fullname: Li, Dangsheng – sequence: 9 givenname: Jinsong surname: Li fullname: Li, Jinsong
BackLink	https://www.ncbi.nlm.nih.gov/pubmed/24315440$$D View this record in MEDLINE/PubMed
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PublicationTitle	Cell stem cell
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Snippet	The CRISPR-Cas9 system has been employed to generate mutant alleles in a range of different organisms. However, so far there have not been reports of use of...
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SubjectTerms	Animals Base Sequence Cataract - genetics Cataract - therapy Clustered Regularly Interspaced Short Palindromic Repeats - genetics CRISPR-Associated Proteins - metabolism Disease Models, Animal gamma-Crystallins - genetics gamma-Crystallins - therapeutic use Genetic Therapy Mice Molecular Sequence Data RNA, Messenger - genetics RNA, Messenger - metabolism
Title	Correction of a genetic disease in mouse via use of CRISPR-Cas9
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