Abstract:
:Monogenic diseases are often severe, life-threatening disorders for which lifelong palliative treatment is the only option. Over the last two decades, a number of strategies have been devised with the aim to treat these diseases with a genetic approach. Gene therapy has been under development for many years, yet suffers from the lack of an effective and safe vector for the delivery of genetic material into cells. More recently, gene targeting by homologous recombination has been proposed as a safer treatment, by specifically correcting disease-causing mutations. However, low efficiency is a major drawback. The emergence of two technologies could overcome some of these obstacles. Terminally differentiated somatic cells can be reprogrammed, using defined factors, to become induced pluripotent stem cells (iPSCs), which can undergo efficient gene mutation correction with the aid of fusion proteins known as zinc finger nucleases (ZFNs). The amalgamation of these two technologies has the potential to break through the current bottleneck in gene therapy and gene targeting.
journal_name
Biotechnol Advjournal_title
Biotechnology advancesauthors
Wong GK,Chiu ATdoi
10.1016/j.biotechadv.2010.05.019subject
Has Abstractpub_date
2010-11-01 00:00:00pages
715-24issue
6eissn
0734-9750issn
1873-1899pii
S0734-9750(10)00070-4journal_volume
28pub_type
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2010.08.007
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journal_title:Biotechnology advances
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2009.07.002
更新日期:2009-11-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2015.08.001
更新日期:2015-12-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
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更新日期:2018-01-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2012.12.001
更新日期:2013-03-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2013.08.019
更新日期:2014-01-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章
doi:10.1016/0734-9750(93)90016-g
更新日期:1993-01-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2018.07.008
更新日期:2018-11-15 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2019.05.001
更新日期:2019-11-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2013.08.018
更新日期:2014-01-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2015.05.008
更新日期:2015-11-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2016.06.007
更新日期:2016-11-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2010.11.007
更新日期:2011-03-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
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更新日期:2020-12-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章
doi:10.1016/j.biotechadv.2009.04.008
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2015.10.013
更新日期:2015-12-01 00:00:00
abstract::Microalgae are regarded as a potential biomass source for biofuel purposes. With regard to bioethanol production, microalgae seem to overcome traditional substrate drawbacks. Enzymatic activities are responsible for carbon allocation and hence for carbohydrate profiles. Enzyme activities may be manipulated by metaboli...
journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2012.07.003
更新日期:2012-11-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2013.05.005
更新日期:2013-12-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2018.07.003
更新日期:2018-11-15 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2016.10.001
更新日期:2016-12-01 00:00:00
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journal_title:Biotechnology advances
pub_type: 杂志文章,评审
doi:10.1016/j.biotechadv.2016.01.006
更新日期:2016-05-01 00:00:00
abstract::Bovine chymosin, an aspartyl protease extracted from abomasum of suckling calves, is synthesized in vivo as preprochymosin and secreted as prochymosin which is autocatalytically activated to chymosin. Chymosin is bilobular, with Asp 32 and Asp 215 acting as the catalytic residues. Chymosin A and chymosin B have pH opt...
journal_title:Biotechnology advances
pub_type: 杂志文章
doi:10.1016/s0734-9750(99)00010-5
更新日期:1999-09-01 00:00:00