AAV vectors: The Rubik's cube of human gene therapy

A Pupo, A Fernández, SH Low, A François… - Molecular Therapy, 2022 - cell.com
Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Exosome-mediated delivery of gene vectors for gene therapy

L Duan, L Xu, X Xu, Z Qin, X Zhou, Y Xiao, Y Liang… - Nanoscale, 2021 - pubs.rsc.org
Gene vectors are nucleic acids that carry genetic materials or gene editing devices into cells
to exert the sustained production of therapeutic proteins or to correct erroneous genes of the …

Targeted therapy using engineered extracellular vesicles: principles and strategies for membrane modification

Q Liu, D Li, X Pan, Y Liang - Journal of nanobiotechnology, 2023 - Springer
Abstract Extracellular vesicles (EVs) are 30–150 nm membrane-bound vesicles naturally
secreted by cells and play important roles in intercellular communication by delivering …

Viral Vectors, Exosomes, and Vexosomes: Potential armamentarium for delivering CRISPR/Cas to cancer cells

M Farzanehpour, A Miri, AG Alvanegh… - Biochemical …, 2023 - Elsevier
The underlying cause of cancer is genetic disruption, so gene editing technologies,
particularly CRISPR/Cas systems can be used to go against cancer. The field of gene …

Vector engineering, strategies and targets in cancer gene therapy

V Singh, N Khan, GR Jayandharan - Cancer Gene Therapy, 2022 - nature.com
Understanding the molecular basis of disease and the design of rationally designed
molecular therapies has been the holy grail in the management of human cancers. Gene …

Roles of small extracellular vesicles in the development, diagnosis and possible treatment strategies for hepatocellular carcinoma

S Yang, J Wang, S Wang… - … journal of oncology, 2022 - spandidos-publications.com
Hepatocellular carcinoma (HCC) is the most common malignancy of hepatocytes accounting
for 75‑85% of primary hepatic carcinoma cases. Small extracellular vesicles (sEVs) …

Targeting non-coding RNA for glioblastoma therapy: the challenge of overcomes the blood-brain barrier

RK Sharma, C Calderon… - Frontiers in Medical …, 2021 - frontiersin.org
Glioblastoma (GBM) is the most malignant form of all primary brain tumors, and it is
responsible for around 200,000 deaths each year worldwide. The standard therapy for GBM …

The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution

A Kachanov, A Kostyusheva, S Brezgin… - Medicinal Research …, 2024 - Wiley Online Library
Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …

Extracellular vesicles as novel drug-delivery systems through intracellular communications

Y Matsuzaka, R Yashiro - Membranes, 2022 - mdpi.com
Since it has been reported that extracellular vesicles (EVs) carry cargo using cell-to-cell
comminication according to various in vivo situations, they are exprected to be applied as …

Chemically modified extracellular vesicles and applications in radiolabeling and drug delivery

ER N'Diaye, NS Orefice, C Ghezzi, A Boumendjel - Pharmaceutics, 2022 - mdpi.com
Extracellular vesicles (EVs) have been exploited as bio-inspired drug delivery systems
(DDS) in the biomedical field. EVs have more advantages than synthetic nanoparticles: they …