Fanconi anemia gene editing by the CRISPR/Cas9 system
Genome engineering with designer nucleases is a rapidly progressing field, and the ability to correct human gene mutations in situ is highly desirable. We employed fibroblasts derived from a patient with Fanconi anemia as a model to test the ability of the clustered regularly interspaced short palin...
Gespeichert in:
| Hauptverfasser: | , , , |
|---|---|
| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
December 3, 2014
|
| In: |
Human gene therapy
Year: 2014, Jahrgang: 26, Heft: 2, Pages: 114-126 |
| ISSN: | 1557-7422 |
| DOI: | 10.1089/hum.2014.111 |
| Online-Zugang: | Verlag, Volltext: http://dx.doi.org/10.1089/hum.2014.111 Verlag, Volltext: http://online.liebertpub.com/doi/abs/10.1089/hum.2014.111 
|
| Verfasserangaben: | Mark J. Osborn, Richard Gabriel, Beau R. Webber, Anthony P. DeFeo, Amber N. McElroy, Jordan Jarjour, Colby G. Starker, John E. Wagner, J. Keith Joung, Daniel F. Voytas, Christof von Kalle, Manfred Schmidt, Bruce R. Blazar, Jakub Tolar |
MARC
| LEADER | 00000caa a2200000 c 4500 | ||
|---|---|---|---|
| 001 | 1557857385 | ||
| 003 | DE-627 | ||
| 005 | 20230427134611.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 170503s2014 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1089/hum.2014.111 |2 doi | |
| 035 | |a (DE-627)1557857385 | ||
| 035 | |a (DE-576)487857380 | ||
| 035 | |a (DE-599)BSZ487857380 | ||
| 035 | |a (OCoLC)1340973039 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rda | ||
| 041 | |a eng | ||
| 084 | |a 33 |2 sdnb | ||
| 100 | 1 | |a Osborn, Mark J. |e VerfasserIn |0 (DE-588)1131174356 |0 (DE-627)885480430 |0 (DE-576)48785313X |4 aut | |
| 245 | 1 | 0 | |a Fanconi anemia gene editing by the CRISPR/Cas9 system |c Mark J. Osborn, Richard Gabriel, Beau R. Webber, Anthony P. DeFeo, Amber N. McElroy, Jordan Jarjour, Colby G. Starker, John E. Wagner, J. Keith Joung, Daniel F. Voytas, Christof von Kalle, Manfred Schmidt, Bruce R. Blazar, Jakub Tolar |
| 264 | 1 | |c December 3, 2014 | |
| 300 | |a 13 | ||
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Gesehen am 03.05.2017 | ||
| 520 | |a Genome engineering with designer nucleases is a rapidly progressing field, and the ability to correct human gene mutations in situ is highly desirable. We employed fibroblasts derived from a patient with Fanconi anemia as a model to test the ability of the clustered regularly interspaced short palindromic repeats/Cas9 nuclease system to mediate gene correction. We show that the Cas9 nuclease and nickase each resulted in gene correction, but the nickase, because of its ability to preferentially mediate homology-directed repair, resulted in a higher frequency of corrected clonal isolates. To assess the off-target effects, we used both a predictive software platform to identify intragenic sequences of homology as well as a genome-wide screen utilizing linear amplification-mediated PCR. We observed no off-target activity and show RNA-guided endonuclease candidate sites that do not possess low sequence complexity function in a highly specific manner. Collectively, we provide proof of principle for precision genome editing in Fanconi anemia, a DNA repair-deficient human disorder. | ||
| 700 | 1 | |a Gabriel, Richard |e VerfasserIn |0 (DE-588)1017959846 |0 (DE-627)690473427 |0 (DE-576)354092332 |4 aut | |
| 700 | 1 | |a Kalle, Christof von |d 1962- |e VerfasserIn |0 (DE-588)1036481115 |0 (DE-627)75107926X |0 (DE-576)168957396 |4 aut | |
| 700 | 1 | |a Schmidt, Manfred |d 1967-2022 |e VerfasserIn |0 (DE-588)122922301 |0 (DE-627)706092430 |0 (DE-576)293477620 |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Human gene therapy |d New York, NY : Liebert, 1990 |g 26(2014), 2, Seite 114-126 |h Online-Ressource |w (DE-627)306585332 |w (DE-600)1498064-2 |w (DE-576)094476950 |x 1557-7422 |7 nnas |a Fanconi anemia gene editing by the CRISPR/Cas9 system |
| 773 | 1 | 8 | |g volume:26 |g year:2014 |g number:2 |g pages:114-126 |g extent:13 |a Fanconi anemia gene editing by the CRISPR/Cas9 system |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1089/hum.2014.111 |x Verlag |x Resolving-System |3 Volltext |
| 856 | 4 | 0 | |u http://online.liebertpub.com/doi/abs/10.1089/hum.2014.111 |x Verlag |3 Volltext |
| 951 | |a AR | ||
| 992 | |a 20170503 | ||
| 993 | |a Article | ||
| 994 | |a 2014 | ||
| 998 | |g 122922301 |a Schmidt, Manfred |m 122922301:Schmidt, Manfred |d 140000 |e 140000PS122922301 |k 0/140000/ |p 12 | ||
| 998 | |g 1036481115 |a Kalle, Christof von |m 1036481115:Kalle, Christof von |d 910000 |e 910000PK1036481115 |k 0/910000/ |p 11 | ||
| 998 | |g 1017959846 |a Gabriel, Richard |m 1017959846:Gabriel, Richard |d 140000 |e 140000PG1017959846 |k 0/140000/ |p 2 | ||
| 999 | |a KXP-PPN1557857385 |e 2968502042 | ||
| BIB | |a Y | ||
| SER | |a journal | ||
| JSO | |a {"relHost":[{"recId":"306585332","disp":"Fanconi anemia gene editing by the CRISPR/Cas9 systemHuman gene therapy","origin":[{"publisher":"Liebert","dateIssuedDisp":"1990-","dateIssuedKey":"1990","publisherPlace":"New York, NY"}],"language":["eng"],"title":[{"title_sort":"Human gene therapy","title":"Human gene therapy"}],"physDesc":[{"extent":"Online-Ressource"}],"part":{"volume":"26","text":"26(2014), 2, Seite 114-126","year":"2014","pages":"114-126","extent":"13","issue":"2"},"type":{"bibl":"periodical","media":"Online-Ressource"},"id":{"eki":["306585332"],"zdb":["1498064-2"],"issn":["1557-7422"]},"pubHistory":["1.1990 -"]}],"note":["Gesehen am 03.05.2017"],"physDesc":[{"extent":"13 S."}],"type":{"bibl":"article-journal","media":"Online-Ressource"},"id":{"eki":["1557857385"],"doi":["10.1089/hum.2014.111"]},"language":["eng"],"title":[{"title_sort":"Fanconi anemia gene editing by the CRISPR/Cas9 system","title":"Fanconi anemia gene editing by the CRISPR/Cas9 system"}],"person":[{"role":"aut","family":"Osborn","display":"Osborn, Mark J.","given":"Mark J."},{"family":"Gabriel","display":"Gabriel, Richard","given":"Richard","role":"aut"},{"role":"aut","family":"Kalle","display":"Kalle, Christof von","given":"Christof von"},{"given":"Manfred","display":"Schmidt, Manfred","family":"Schmidt","role":"aut"}],"recId":"1557857385","name":{"displayForm":["Mark J. Osborn, Richard Gabriel, Beau R. Webber, Anthony P. DeFeo, Amber N. McElroy, Jordan Jarjour, Colby G. Starker, John E. Wagner, J. Keith Joung, Daniel F. Voytas, Christof von Kalle, Manfred Schmidt, Bruce R. Blazar, Jakub Tolar"]},"origin":[{"dateIssuedDisp":"December 3, 2014","dateIssuedKey":"2014"}]} | ||
| SRT | |a OSBORNMARKFANCONIANE3201 | ||