CRISPR-Cas İmmün Sisteminin Biyolojisi, Mekanizması ve Kullanım Alanları

Year 2016, Volume: 8 Issue: 2, 11 - 21, 15.06.2016

Zehra Gün Gök Beste Çağdaş Tunalı

https://doi.org/10.29137/umagd.346148

Cited By: 1

Abstract

Hedeflenmiş nükleazlar genom
düzenlenmesinde yaygın olarak kullanılmaktadır. Kısa süre önce, düzenli
aralıklarla bölünmüş palindromik tekrar kümeleri (CRISPRs)-ilişkili Cas9
nükleazları genom düzenleme çalışmalarında ilk defa kullanılmış ve o zamandan
beri bu alanda devrim yaratmıştır. CRISPR-Cas9 genom düzenleme aracının bu
büyük başarısının ardında Cas9’u istenilen DNA lokusuna hedefleyen kılavuz
RNA’nın tasarımının basitliği ve CRISPR-Cas9 aracılı DNA kırılmalarının yüksek
özgüllük ve verimlilikte olması yatmaktadır. Yakın zamanda yapılan bazı
çalışmalarda, in vivo hayvan
modellerinde ve ex vivo somatik ve
uyarılmış pluripotent kök hücrelerinde hastalığa neden olan allellerin
düzenlenmesinde CRISPR-Cas9 sistemi başarıyla kullanılarak terapötik genom
düzenlenmesinin klinik uygulamaları için umutları arttırmıştır. Bu derleme ile
bu sistemlerden en çok kullanılan CRISPR-Cas9 Tip II sisteminin diğer
sistemlere göre avantaj ve dezavantajları belirtilmiş ve bu sistemler ile
yapılan uygulamalara değinilmiştir. Ayrıca bu derlemede, çeşitli araştırma ya
da translasyonel uygulamalarda kullanılan ve olağanüstü bir mikrobiyal savunma
sisteminden türetilen CRISPR-Cas9’un gelişiminden ve uygulamalarındaki
zorluklarından bahsedilmiştir.

Keywords

CRISPR, CRISPR-Cas9 Tip II sistem, genom düzenleme, Cas proteinleri, HDR, NHEJ

Biology, Mechanism and Applications of CRISPR-Cas Immune System

Abstract

Targeted nucleases are widely
used as tools for genome editing. Recently, the clustered regularly interspaced
short palindromic repeat (CRISPR)-associated Cas9 nuclease was used in the
genome editing studies for the first time, and since then has largely revolutionized
the field. The great success of the CRISPR/Cas9 genome editing tool is powered
by the simple design principle of the guide RNA that aims Cas9 to the target
DNA locus, and by the high specificity and efficiency of CRISPR/Cas9-generated
DNA breaks. Several studies lately used CRISPR-Cas9 to successfully arrange
disease-causing alleles in vivo in
animal models and ex vivo in somatic
and induced pluripotent stem cells, increasing hope for therapeutic genome
editing in the clinics. In this study, we focus on the CRISPR-Cas9 Type II
system, provide specific examples for use of the system, and highlight the
advantages and disadvantages of CRISPR versus other techniques. Also in this
review, we briefly describe the development and applications of Cas9 which is
derived from a remarkable microbial defense system for a variety of research or
translational applications while highlighting challenges.

Keywords

CRISPR, CRISPR-Cas9 Type II system, genome editing, Cas proteins, HDR, NHEJ

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