Non-viral delivery of the CRISPR/Cas system: DNA versus RNA versus RNP

Y Lin, E Wagner, U Lächelt - Biomaterials Science, 2022 - pubs.rsc.org
Since its discovery, the CRISPR/Cas technology has rapidly become an essential tool in
modern biomedical research. The opportunities to specifically modify and correct genomic …

Harnessing nanoparticles for the efficient delivery of the CRISPR/Cas9 system

H Rahimi, M Salehiabar, J Charmi, M Barsbay… - Nano Today, 2020 - Elsevier
Exploiting bacterial DNA-acting enzymes and expanding their repertoire for genome
engineering have been major technological and conceptual advances in molecular biology …

[HTML][HTML] Recent advances in genome editing of stem cells for drug discovery and therapeutic application

J Lee, D Bayarsaikhan, G Bayarsaikhan, JS Kim… - Pharmacology & …, 2020 - Elsevier
Genome engineering technologies right from viral vector-mediated to protein-based editing—
which include zinc finger nucleases, TALENs, and CRISPR/Cas systems—have been …

Hematopoietic stem cell gene therapy: The optimal use of lentivirus and gene editing approaches

A Lamsfus-Calle, A Daniel-Moreno, G Urena-Bailen… - Blood reviews, 2020 - Elsevier
Due to pioneering in vitro investigations on gene modification, gene engineering platforms
have incredibly improved to a safer and more powerful tool for the treatment of multiple …

Genome-wide specificity of dCpf1 cytidine base editors

D Kim, K Lim, D Kim, JS Kim - Nature Communications, 2020 - nature.com
Cpf1-linked base editors broaden the targeting scope of programmable cytidine deaminases
by recognizing thymidine-rich protospacer-adjacent motifs (PAM) without inducing DNA …

CRISPR-generated animal models of duchenne muscular dystrophy

KRQ Lim, Q Nguyen, K Dzierlega, Y Huang, T Yokota - Genes, 2020 - mdpi.com
Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disorder
most commonly caused by mutations disrupting the reading frame of the dystrophin (DMD) …

Advances in genome editing: The technology of choice for precise and efficient β-thalassemia treatment

G Ali, MA Tariq, K Shahid, FJ Ahmad, J Akram - Gene therapy, 2021 - nature.com
Beta (β)-thalassemia is one of the most significant hemoglobinopathy worldwide. The high
prevalence of the β-thalassemia carriers aggravates the disease burden for patients and …

Recent updates on corticosteroid-induced neuropsychiatric disorders and theranostic advancements through gene editing tools

M Singh, V Agarwal, D Jindal, P Pancham, S Agarwal… - Diagnostics, 2023 - mdpi.com
The vast use of corticosteroids (CCSs) globally has led to an increase in CCS-induced
neuropsychiatric disorders (NPDs), a very common manifestation in patients after CCS …

Ethics assessment in research proposals adopting CRISPR technology

F Hirsch, R Iphofen, Z Koporc - Biochemia Medica, 2019 - hrcak.srce.hr
Sažetak The rapid and exponential growth of genome editing has posed many challenges
for bioethics. This article briefly explains the nature of the technique and the particularly …

CRISPR-Cas gene knockouts to optimize engineered T cells for cancer immunotherapy

V De Castro, J Galaine, R Loyon, Y Godet - Cancer Gene Therapy, 2024 - nature.com
Abstract While CAR-T and tgTCR-T therapies have exhibited noteworthy and promising
outcomes in hematologic and solid tumors respectively, a set of distinct challenges remains …