[HTML][HTML] Comprehensive review of CRISPR-based gene editing: mechanisms, challenges, and applications in cancer therapy

M Chehelgerdi, M Chehelgerdi… - Molecular cancer, 2024 - Springer
The CRISPR system is a revolutionary genome editing tool that has the potential to
revolutionize the field of cancer research and therapy. The ability to precisely target and edit …

Genetic engineering of T cells for immunotherapy

GI Ellis, NC Sheppard, JL Riley - Nature Reviews Genetics, 2021 - nature.com
Genetically engineered T cell immunotherapies have provided remarkable clinical success
to treat B cell acute lymphoblastic leukaemia by harnessing a patient's own T cells to kill …

Prediction of on-target and off-target activity of CRISPR–Cas13d guide RNAs using deep learning

HH Wessels, A Stirn, A Méndez-Mancilla, EJ Kim… - Nature …, 2024 - nature.com
Transcriptome engineering applications in living cells with RNA-targeting CRISPR effectors
depend on accurate prediction of on-target activity and off-target avoidance. Here we design …

RAD51 bypasses the CMG helicase to promote replication fork reversal

W Liu, Y Saito, J Jackson, R Bhowmick, MT Kanemaki… - Science, 2023 - science.org
Replication fork reversal safeguards genome integrity as a replication stress response. DNA
translocases and the RAD51 recombinase catalyze reversal. However, it remains unknown …

[HTML][HTML] MaveDB: an open-source platform to distribute and interpret data from multiplexed assays of variant effect

D Esposito, J Weile, J Shendure, LM Starita… - Genome biology, 2019 - Springer
Multiplex assays of variant effect (MAVEs), such as deep mutational scans and massively
parallel reporter assays, test thousands of sequence variants in a single experiment. Despite …

Next-generation AAV vectors—do not judge a virus (only) by its cover

C Domenger, D Grimm - Human molecular genetics, 2019 - academic.oup.com
Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

Titrating gene expression using libraries of systematically attenuated CRISPR guide RNAs

M Jost, DA Santos, RA Saunders, MA Horlbeck… - Nature …, 2020 - nature.com
A lack of tools to precisely control gene expression has limited our ability to evaluate
relationships between expression levels and phenotypes. Here, we describe an approach to …

[HTML][HTML] Emerging approaches for regulation and control of CAR T cells: a mini review

LJB Brandt, MB Barnkob, YS Michaels… - Frontiers in …, 2020 - frontiersin.org
Chimeric antigen receptor (CAR) T cells have emerged as a promising treatment for patients
with advanced B-cell cancers. However, widespread application of the therapy is currently …

[HTML][HTML] Context-aware synthetic biology by controller design: Engineering the mammalian cell

N Shakiba, RD Jones, R Weiss, D Del Vecchio - Cell systems, 2021 - cell.com
The rise of systems biology has ushered a new paradigm: the view of the cell as a system
that processes environmental inputs to drive phenotypic outputs. Synthetic biology provides …

Synthetic genetic circuits to uncover the OCT4 trajectories of successful reprogramming of human fibroblasts

K Ilia, N Shakiba, T Bingham, RD Jones… - Science …, 2023 - science.org
Reprogramming human fibroblasts to induced pluripotent stem cells (iPSCs) is inefficient,
with heterogeneity among transcription factor (TF) trajectories driving divergent cell states …