We also discuss the applications of these novel technologies in preclinical and clinical studies in four groups according to the mechanism used, and lastly, summarize the problems in these technologies.
📌 3 Maddede Bu Makale
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Amaç & Hipotez:The development of genome editing technologies has given the chance to researchers to manipulate any genomic sequences precisely.
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Temel Bulgular & Veri:This ability is very useful for creating animal models to study human diseases in vivo; for easy creation of isogenic cell lines to study in vitro and most importantly for overcoming many disadvantages that the researchers faced during the human gene therapy trials.
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Sonuç & Katkı:We also discuss the applications of these novel technologies in preclinical and clinical studies in four groups according to the mechanism used, and lastly, summarize the problems in these technologies.
🔬Tür:Literatür & Derleme Çalışması
📍Kapsam:Genişletilmiş Akademik Örneklem
⚙️Değişkenler:Alan Parametreleri
Özet
The development of genome editing technologies has given the chance to researchers to manipulate any genomic sequences precisely. This ability is very useful for creating animal models to study human diseases in vivo; for easy creation of isogenic cell lines to study in vitro and most importantly for overcoming many disadvantages that the researchers faced during the human gene therapy trials. Here we review the basic mechanisms of genome editing technology and the four genome-editing platforms. We also discuss the applications of these novel technologies in preclinical and clinical studies in four groups according to the mechanism used, and lastly, summarize the problems in these technologies.
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Bu Makaleye Atıf Yap
TUNCAY, F. Y., DİNÇER, P. R. (2018). Genome Editing Technologies: From Bench Side to Bedside. Acta Medica, Cilt: 49 - Sayı: 3, 30–40. https://ajindex.com/articles/144672
Maeder ML, Gersbach CA. Genome-editing Technologies for Gene and Cell Therapy. Mol Ther. 2016; 24(3): 430-46.
Rouet P, Smih F, Jasin M. Expression of a site-specific endonu- clease stimulates homologous recombination in mammalian cells. Proc Natl Acad Sci U S A. 1994; 91(13): 6064-8.
Belfort M, Roberts RJ. Homing endonucleases: keeping the house in order. Nucleic Acids Res. 1997; 25(17): 3379-88.
Bibikova M, Golic M, Golic KG, et al. Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases. Genetics. 2002; 161(3): 1169-75.
Doyon Y, Vo TD, Mendel MC, et al. Enhancing zinc-finger-nu- clease activity with improved obligate heterodimeric archi- tectures. Nat Methods. 2011; 8(1): 74-9.
Boch J, Scholze H, Schornack S, et al. Breaking the code of DNA binding specificity of TAL-type III effectors. Science. 2009; 326(5959): 1509-12.
Boissel S, Jarjour J, Astrakhan A, et al. megaTALs: a rare-cleav- ing nuclease architecture for therapeutic genome engineer- ing. Nucleic Acids Res. 2014; 42(4): 2591-601.
Jore MM, Lundgren M, van Duijn E, et al. Structural basis for CRISPR RNA-guided DNA recognition by Cascade. Nat Struct Mol Biol. 2011; 18(5): 529-36. [9] Jinek M, Chylinski K, Fonfara I, et al. Science. A programma- ble dual-RNA-guided DNA endonuclease in adaptive bacteri- al immunity. 2012; 337(6096): 816-21.
Zetsche B, Gootenberg JS, Abudayyeh OO, et al. Cpf1 is a sin- gle RNA-guided endonuclease of a class 2 CRISPR-Cas sys- tem. Cell. 2015 Oct 22;163(3):759-71.
Ran FA, Cong L, Yan WX, et al. In vivo genome editing using Staphylococcus aureus Cas9. Nature. 2015; 520(7546): 186-91.
Kleinstiver BP, Prew MS, Tsai SQ, et al. Engineered CRISPR- Cas9 nucleases with altered PAM specificities. Nature. 2015; 523(7561): 481-5.
Takata M, Sasaki MS, Sonoda E, et al. Homologous recombi- nation and non-homologous end-joining pathways of DNA double-strand break repair have overlapping roles in the maintenance of chromosomal integrity in vertebrate cells. EMBO J. 1998; 17(18): 5497-508.
Gaj T, Gersbach CA, Barbas CF. ZFN, TALEN, and CRISPR/Cas- based methods for genome engineering. Trends Biotechnol. 2013; 31(7): 397-405.
Canver MC, Bauer DE, Dass A, et al. Characterization of genomic deletion efficiency mediated by clustered regularly interspaced pa