Non-viral vectors in gene therapy: recent development, challenges, and prospects

H Zu, D Gao - The AAPS journal, 2021 - Springer
Gene therapy has been experiencing a breakthrough in recent years, targeting various
specific cell groups in numerous therapeutic areas. However, most recent clinical studies …

Efficient peptide-mediated in vitro delivery of Cas9 RNP

O Gustafsson, J Rädler, S Roudi, T Lehto, M Hällbrink… - Pharmaceutics, 2021 - mdpi.com
The toolbox for genetic engineering has quickly evolved from CRISPR/Cas9 to a myriad of
different gene editors, each with promising properties and enormous clinical potential …

Integrating pharmacogenetics, multi‐omics and machine learning in the novel therapeutic view of pulmonary hypertension

C Li, J Xue, D Zhou, X Qi, T Chen… - Interdisciplinary …, 2025 - Wiley Online Library
It is a complex disease with multiple pathogenesis that causes pulmonary hypertension
(PH). In addition to the commonly used drugs for treating PH, pharmacogenomic therapies …

Development of non-viral vectors for neuronal-targeted delivery of CRISPR-Cas9 RNA-proteins as a therapeutic strategy for neurological disorders

KE Shalaby, M Aouida, V Gupta, H Abdesselem… - Biomaterials …, 2022 - pubs.rsc.org
The aging population contributes to an increase in the prevalence of neurodegenerative
diseases, such as Parkinson's disease (PD). Due to the progressive nature of these …

Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides

JP Bost, M Ojansivu, MJ Munson, E Wesén… - Communications …, 2022 - nature.com
The therapeutic and research potentials of oligonucleotides (ONs) have been hampered in
part by their inability to effectively escape endosomal compartments to reach their cytosolic …

Non-viral gene delivery systems

H Faneca - Pharmaceutics, 2021 - mdpi.com
The advances in the field of gene therapy have significantly improved the possibility for
nucleic acids as highly promising agents for the treatment of both inherited and acquired …

Internalisation and biological activity of nucleic acids delivering cell-penetrating peptide nanoparticles is controlled by the biomolecular corona

A Lorents, M Maloverjan, K Padari, M Pooga - Pharmaceuticals, 2021 - mdpi.com
Nucleic acid molecules can be transferred into cells to alter gene expression and, thus,
alleviate certain pathological conditions. Cell-penetrating peptides (CPPs) are vectors that …

Lipophilic peptide dendrimers for delivery of splice-switching oligonucleotides

H Daralnakhla, O Saher, S Zamolo, S Bazaz, J P. Bost… - Pharmaceutics, 2021 - mdpi.com
Non-viral transfection reagents are continuously being developed in attempt to replace viral
vectors. Among those non-viral vectors, dendrimers have gained increasing interest due to …

Application of lyophilized gene-delivery formulations to dental implant surfaces: non-cariogenic lyoprotectant preserves transfection activity of polyplexes long-term

WI Malkawi, NZ Laird, P Phruttiwanichakun… - Journal of …, 2023 - Elsevier
Titanium is the metal of choice for dental implants because of its biocompatibility and ability
to merge with human bone tissue. Despite the great success rate of dental implants, early …

Methods for CPP Functionalization with Oligonucleotides

Ü Langel - CPP, Cell-Penetrating Peptides, 2023 - Springer
This Chapter summarizes the methods for CPP-conjugated oligonucleotide-based agents
with the potential to use as the therapeutic drugs. The methods for gene silencing (or …