Nanotechnology-Based Strategies to Overcome Current Barriers in Gene Delivery.

Sofía Mirón-Barroso,Elena B. Domènech,Sonia Trigueros

doi:10.3390/ijms22168537

Abstract

Nanomaterials are currently being developed for the specific cell/tissue/organ delivery of genetic material. Nanomaterials are considered as non-viral vectors for gene therapy use. However, there are several requirements for developing a device small enough to become an efficient gene-delivery tool. Considering that the non-viral vectors tested so far show very low efficiency of gene delivery, there is a need to develop nanotechnology-based strategies to overcome current barriers in gene delivery. Selected nanostructures can incorporate several genetic materials, such as plasmid DNA, mRNA, and siRNA. In the field of nanotechnologies, there are still some limitations yet to be resolved for their use as gene delivery systems, such as potential toxicity and low transfection efficiency. Undeniably, novel properties at the nanoscale are essential to overcome these limitations. In this paper, we will explore the latest advances in nanotechnology in the gene delivery field.

Highlights

Nanomaterials are currently being developed for the specific cell/tissue/organ delivery of genetic material
We will explore the latest advances in nanotechnology in the gene delivery field
The major RNA therapeutic methods are: (I) antisense oligonucleotides (AONs) which are small RNA or DNA chemically modified molecules that bind by complementary base pair to the pre-mRNA and their main functions are to exclude exons and pseudo-exons, include exons, degrade transcripts and block the translation [13]; (II) U1 spliceosomal

Summary

Introduction

Numerous diseases find their roots at the genetic level. The human genome project and the latest advances in molecular genetics and high throughput techniques allowed us to understand the genetic basis of many pathologies and, to identify new therapeutic targets [1,2]. New strategies are being developed for “undruggable” diseases [3]. In the past few decades, gene therapy emerged as a potential treatment for a wide range of diseases, including cancer, cardiovascular and neurological diseases [4]. Due to the diseases and genetic defects’ heterogeneity, different molecular approaches have been developed to achieve the therapeutic goal. Publisher’s Note: MDPI stays neutral with regard to jurisdictional claims in published maps and institutional affiliations

Gene Therapy

Gene Editing

Genedepends

RNA Therapy

Why Does Gene Therapy Need a Carrier?

Viral Vectors

Physical Methods

Organic Strategies

Inorganic Strategies

Protein Corona

Active and Passive Targeting

Internalization and Intracellular Trafficking