Biomaterial‐based gene therapy

Abstract

Gene therapy, a medical approach that involves the correction or replacement of defective and abnormal genes, plays an essential role in the treatment of complex and refractory diseases, such as hereditary diseases, cancer, and rheumatic immune diseases. Nucleic acids alone do not easily enter the target cells due to their easy degradation in vivo and the structure of the target cell membranes. The introduction of genes into biological cells is often dependent on gene delivery vectors, such as adenoviral vectors, which are commonly used in gene therapy. However, traditional viral vectors have strong immunogenicity while also presenting a potential infection risk. Recently, biomaterials have attracted attention for use as efficient gene delivery vehicles, because they can avoid the drawbacks associated with viral vectors. Biomaterials can improve the biological stability of nucleic acids and the efficiency of intracellular gene delivery. This review is focused on biomaterial‐based delivery systems in gene therapy and disease treatment. Herein, we review the recent developments and modalities of gene therapy. Additionally, we discuss nucleic acid delivery strategies, with a focus on biomaterial‐based gene delivery systems. Furthermore, the current applications of biomaterial‐based gene therapy are summarized.

Nucleic Acid Delivery Nanotechnologies for In Vivo Cell Programming

“…A major characteristic of using viral vectors is that they can persist in the body for relatively long periods of time and therefore have the potential to remain highly efficacious. , Regulatory elements, which are DNA sequences that promote or enhance gene expression, can be selected to control and drive high levels of transgene expression . However, current gene therapies lack the ability to be controlled or turned off after administration and could contribute to toxicity if delivered to off-target cells. , Viral vectors are also naturally capable of performing endosomal escape as they evolved to do so in order to infect cells and release their genetic cargo.…”

Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

“…The creation of risk-free and efficient gene-editing medicines is being facilitated by developments in biomaterial design, formulation optimization, and safety evaluations. Preclinical research in animal models has shown encouraging findings, suggesting the possibility of clinical use in the future ( Bashor et al, 2022 ; Yu et al, 2023b ).…”

Developing hydrogels for gene therapy and tissue engineering

“…Gene therapy is the introduction of genes into cells to up-regulate the expression of target genes to treat or repair tissue defects [ 2 , 3 ]. It exhibits high specificity, low immune rejection, and durability; it is widely used in the treatment of cystic fibrosis, hereditary retinopathy, multiple sclerosis, bone defect repair, and cancer [ 4 , 5 ].…”