Polymer-Based Transfection Agents Used in CRISPR-CAS9 System

Abstract

Genome editing is a method used to make desired changes in the target gene. Today, various methods are used for genome-editing studies; among them, one of the most widely used methods is the clustered, regularly interspaced short palindromic repeats (CRISPR). CRISPR-associated (Cas) genes and their corresponding CRISPR sequences constitute CRISPR-Cas systems. Due to its simplicity, it is likely that the CRISPR–Cas system could be used effectively in ex vivo gene therapy studies in humans. If this happens, the importance of CRISPR carrier systems will gradually increase. Viral and non-viral systems are used as delivery modalities in genome-editing studies. It has been proven that nanoparticles are the most promising tools for gene therapy due to their adjustable size, surface, shape, and biological behaviours. The polymeric carrier system has become the main non-viral substitute for gene delivery due to its reduced immunogenicity and pathogenicity. In this review, information about current studies related to polymeric carriers used in non-viral CRISPR delivery systems is presented.

Keywords

• CRISPR
• non-viraltransfection agent
• polymeric carrier system

CRISPR-CAS9 Sisteminde Kullanılan Polimer Bazlı Transfeksiyon Ajanları

Öz

Genom düzenleme, hedef gende istenilen değişiklikleri yapmak için kullanılan bir yöntemdir. Günümüzde genom düzenleme çalışmaları için çeşitli yöntemler kullanılmaktadır; bunlar arasında en yaygın kullanılan yöntemlerden biri kümelenmiş, düzenli aralıklarla yerleştirilmiş kısa palindromik tekrarlardır (CRISPR). CRISPR ile ilişkili (Cas) genleri ve bunlara karşılık gelen CRISPR dizileri, CRISPR-Cas sistemlerini oluşturur. Basitliği nedeniyle, CRISPR-Cas sisteminin insanlarda ex vivogen terapisi çalışmalarında etkili bir şekilde kullanılmaya başlanmıştır veCRISPR taşıyıcı sistemlerin önemi giderek artmaktadır.Genom düzenleme çalışmalarında dağıtım yöntemleri olarak viral ve viral olmayan sistemler kullanılmaktadır. Nanopartiküllerin ayarlanabilir boyutları, yüzeyleri, şekilleri ve biyolojik davranışları nedeniyle gen terapisi için en umut verici araçlar olduğukanıtlanmıştır. Polimerik taşıyıcı sistem, azaltılmış immünojenisitesi ve patojenitesi nedeniyle gen aktarımı için viral olmayan ana ikame haline gelmiştir. Bu derlemede, viral olmayan CRISPR dağıtım sistemlerinde kullanılan polimerik taşıyıcılarla ilgiligüncel çalışmalar hakkında bilgiler sunulmaktadır.

Anahtar Kelimeler

• CRISPR
• Viral olmayan Transfeksiyon ajanları
• Polimerik Trasnfeksiyon Ajanları

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