Delivering CRISPR: a review of the challenges and approaches

CA Lino, JC Harper, JP Carney, JA Timlin - Drug delivery, 2018 - Taylor & Francis
Gene therapy has long held promise to correct a variety of human diseases and defects.
Discovery of the Clustered Regularly-Interspaced Short Palindromic Repeats (CRISPR), the …

The sound of silence: Transgene silencing in mammalian cell engineering

A Cabrera, HI Edelstein, F Glykofrydis, KS Love… - Cell Systems, 2022 - cell.com
To elucidate principles operating in native biological systems and to develop novel
biotechnologies, synthetic biology aims to build and integrate synthetic gene circuits within …

High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition

S Selvaraj, WN Feist, S Viel, S Vaidyanathan… - Nature …, 2024 - nature.com
Therapeutic applications of nuclease-based genome editing would benefit from improved
methods for transgene integration via homology-directed repair (HDR). To improve HDR …

Marker-free carotenoid-enriched rice generated through targeted gene insertion using CRISPR-Cas9

OX Dong, S Yu, R Jain, N Zhang, PQ Duong… - Nature …, 2020 - nature.com
Targeted insertion of transgenes at pre-determined plant genomic safe harbors provides a
desirable alternative to insertions at random sites achieved through conventional methods …

Induced pluripotent stem cells (iPSCs)—roles in regenerative therapies, disease modelling and drug screening

MAM Aboul-Soud, AJ Alzahrani, A Mahmoud - Cells, 2021 - mdpi.com
The discovery of induced pluripotent stem cells (iPSCs) has made an invaluable contribution
to the field of regenerative medicine, paving way for identifying the true potential of human …

Enhanced CAR T-cell engineering using non-viral Sleeping Beauty transposition from minicircle vectors

R Monjezi, C Miskey, T Gogishvili, M Schleef… - Leukemia, 2017 - nature.com
Immunotherapy with T cell modified with gamma-retroviral or lentiviral (LV) vectors to
express a chimeric antigen receptor (CAR) has shown remarkable efficacy in clinical trials …

Limitations in the design of chimeric antigen receptors for cancer therapy

S Stoiber, BL Cadilha, MR Benmebarek, S Lesch… - Cells, 2019 - mdpi.com
Cancer therapy has entered a new era, transitioning from unspecific chemotherapeutic
agents to increasingly specific immune-based therapeutic strategies. Among these, chimeric …

Harnessing eukaryotic retroelement proteins for transgene insertion into human safe-harbor loci

X Zhang, B Van Treeck, CA Horton, JJR McIntyre… - Nature …, 2024 - nature.com
Current approaches for inserting autonomous transgenes into the genome, such as CRISPR–
Cas9 or virus-based strategies, have limitations including low efficiency and high risk of …

First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B

P Harmatz, CE Prada, BK Burton, H Lau, CM Kessler… - Molecular Therapy, 2022 - cell.com
Zinc-finger nuclease (ZFN)-based in vivo genome editing is a novel treatment that can
potentially provide lifelong protein replacement with single intravenous administration …

Connecting past and present: single-cell lineage tracing

C Chen, Y Liao, G Peng - Protein & Cell, 2022 - academic.oup.com
Central to the core principle of cell theory, depicting cells' history, state and fate is a
fundamental goal in modern biology. By leveraging clonal analysis and single-cell RNA-seq …