Abstract:
:Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) technology has revolutionized therapeutic gene editing by providing researchers with a new method to study and cure diseases previously considered untreatable. While the full range and power of CRISPR technology for therapeutics is being elucidated through in vitro studies, translation to in vivo studies is slow. To date there is no totally effective delivery strategy to carry CRISPR components to the target site in vivo. The complexity of in vivo delivery is furthered by the number of potential delivery methods, the different forms in which CRISPR can be delivered as a therapeutic, and the disease target and tissue type in question. There are major challenges and limitations to delivery strategies, and it is imperative that future directions are guided by well-conducted studies that consider the full effect these variables have on the eventual outcome. In this review we will discuss the advances of the latest in vivo CRISPR/Cas9 delivery strategies and highlight the challenges yet to be overcome.
journal_name
Biol Pharm Bulljournal_title
Biological & pharmaceutical bulletinauthors
Çiçek YA,Luther DC,Kretzmann JA,Rotello VMdoi
10.1248/bpb.b18-00811subject
Has Abstractpub_date
2019-01-01 00:00:00pages
304-311issue
3eissn
0918-6158issn
1347-5215journal_volume
42pub_type
杂志文章,评审abstract::Here, we confirmed that stable expression of B-cell lymphoma-xL (Bcl-xL) in N18TG neuroglioma cells could suppress c-Jun N-terminal protein kinase (JNK) activation, nuclear fragmentation, and cell death caused by etoposide treatment. Moreover, additional overexpression of JNK1 led to partially antagonize the antiapopt...
journal_title:Biological & pharmaceutical bulletin
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journal_title:Biological & pharmaceutical bulletin
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章,评审
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journal_title:Biological & pharmaceutical bulletin
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journal_title:Biological & pharmaceutical bulletin
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abstract::Cilnidipine is a 1,4-dihydropyridine-derived voltage-dependent calcium channel (VDCC) blocker and suppresses N-type VDCC currents in addition to L-type VDCC currents. An earlier investigation has suggested that intrathecally injected cilnidipine produces antinociception by blocking N-type VDCCs in mice. The present st...
journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
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更新日期:2009-10-01 00:00:00
abstract::Recently we showed significance of biosurfactants in the field of non-viral vectors for gene transfection. There, a biosurfactant, mannosylerythritol lipid A (MEL-A), especially increased the efficiency of gene transfection mediated with cationic liposomes. However, the molecular mechanism has not been well-understood...
journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
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pub_type: 杂志文章
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.21.191
更新日期:1998-02-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.24.634
更新日期:2001-06-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.29.1300
更新日期:2006-06-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.31.2321
更新日期:2008-12-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.b15-00489
更新日期:2015-01-01 00:00:00
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更新日期:2002-03-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.b15-00358
更新日期:2016-01-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
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journal_title:Biological & pharmaceutical bulletin
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journal_title:Biological & pharmaceutical bulletin
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章,多中心研究,随机对照试验
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
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abstract::The prophylactic action of scymnol in a rat peripheral arterial occlusion model, involving injection of 5% lactic acid into the femoral artery, was investigated. Increases in serum lactate dehydrogenase (LDH), glutamic oxaloacetic transaminase (GOT) and glutamic pyruvic transaminase (GPT) activities and in plasma leve...
journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.22.822
更新日期:1999-08-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
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更新日期:2009-04-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.17.668
更新日期:1994-05-01 00:00:00
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journal_title:Biological & pharmaceutical bulletin
pub_type: 杂志文章
doi:10.1248/bpb.34.1684
更新日期:2011-01-01 00:00:00