[HTML][HTML] RNA Structure: Past, Future, and Gene Therapy Applications

WA Haseltine, K Hazel, R Patarca - International Journal of Molecular …, 2024 - mdpi.com
First believed to be a simple intermediary between the information encoded in
deoxyribonucleic acid and that functionally displayed in proteins, ribonucleic acid (RNA) is …

Non-clinical safety assessment of novel drug modalities: Genome safety perspectives on viral-, nuclease-and nucleotide-based gene therapies

M Lemmens, L Dorsheimer, A Zeller… - … -Genetic Toxicology and …, 2024 - Elsevier
Gene therapies have emerged as promising treatments for various conditions including
inherited diseases as well as cancer. Ensuring their safe clinical application requires the …

Target-seq: single workflow for detection of genome integration site, DNA translocation and off-target events

PZ Tang, B Ding, C Reyes, D Papp, J Potter - BioTechniques, 2023 - Taylor & Francis
Designed donor DNA delivery through viral or nonviral systems to target loci in the host
genome is a critical step for gene therapy. Adeno-associated virus and lentivirus are leading …

[HTML][HTML] Optimised protocols to generate high titre lentiviral vectors using a novel transfection agent enabling extended HEK293T culture following transient …

S Suleman, S Fawaz, T Roberts, S Ellison… - Journal of Virological …, 2024 - Elsevier
HIV-1 based lentiviral viruses are considered powerful and versatile gene therapy vectors to
deliver therapeutic genes to patients with hereditary or acquired diseases. These vectors …

Engineering of HEK293T Cell Factory for Lentiviral Production by High-Throughput Selected Genes

Z Xinyue, S Li, W Yujie, D Yingcai, B Changhao… - The CRISPR …, 2024 - liebertpub.com
Lentiviral vectors (LVs) are crucial tools in gene therapy and bioproduction, but high-yield
LV production systems are urgently needed. Using clustered regularly interspaced short …