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如何通过控制释放技术来辅助基因传递。

How controlled release technology can aid gene delivery.

机构信息

a Department of Biomaterials, Field of Tissue Engineering, Institute for Frontier Medical Sciences, Kyoto University , 53 Kawara-cho Shogoin, Sakyo-ku Kyoto 606-8507, Japan +81 75 751 4121 ; +81 75 751 4646 ;

出版信息

Expert Opin Drug Deliv. 2015;12(10):1689-701. doi: 10.1517/17425247.2015.1048221. Epub 2015 Jun 29.

Abstract

INTRODUCTION

Many types of gene delivery systems have been developed to enhance the level of gene expression. Controlled release technology is a feasible gene delivery system which enables genes to extend the expression duration by maintaining and releasing them at the injection site in a controlled manner. This technology can reduce the adverse effects by the bolus dose administration and avoid the repeated administration. Biodegradable biomaterials are useful as materials for the controlled release-based gene delivery technology and various biodegradable biomaterials have been developed.

AREAS COVERED

Controlled release-based gene delivery plays a critical role in a conventional gene therapy and genetic engineering. In the gene therapy, the therapeutic gene is released from biodegradable biomaterial matrices around the tissue to be treated. On the other hand, the intracellular controlled release of gene from the sub-micro-sized matrices is required for genetic engineering. Genetic engineering is feasible for cell transplantation as well as research of stem cells biology and medicine.

EXPERT OPINION

DNA hydrogel containing a sequence of therapeutic gene and the exosome including the individual specific nucleic acids may become candidates for controlled release carriers. Technologies to deliver genes to cell aggregates will play an important role in the promotion of regenerative research and therapy.

摘要

简介

为了提高基因表达水平,已经开发出许多类型的基因传递系统。控制释放技术是一种可行的基因传递系统,它可以通过以受控方式在注射部位维持和释放基因来延长基因的表达时间。该技术可以通过减少大剂量给药的不良反应并避免重复给药来降低不良反应。可生物降解的生物材料可用作基于控制释放的基因传递技术的材料,并且已经开发出了各种可生物降解的生物材料。

涵盖的领域

基于控制释放的基因传递在传统的基因治疗和基因工程中起着关键作用。在基因治疗中,治疗基因从待治疗组织周围的可生物降解生物材料基质中释放出来。另一方面,对于基因工程来说,需要从亚微米级基质中对基因进行细胞内控制释放。基因工程对于细胞移植以及干细胞生物学和医学的研究都是可行的。

专家意见

含有治疗基因序列的 DNA 水凝胶和包含个体特定核酸的外泌体可能成为控制释放载体的候选物。将基因递送到细胞聚集体的技术将在促进再生研究和治疗方面发挥重要作用。

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