[HTML][HTML] Strategies to overcome the main challenges of the use of CRISPR/Cas9 as a replacement for cancer therapy

MF Rasul, BM Hussen, A Salihi, BS Ismael, PJ Jalal… - Molecular Cancer, 2022 - Springer
CRISPR/Cas9 (clustered regularly interspaced short palindromic repeats-associated protein
9) shows the opportunity to treat a diverse array of untreated various genetic and …

CRISPR/Cas systems in genome editing: methodologies and tools for sgRNA design, off‐target evaluation, and strategies to mitigate off‐target effects

H Manghwar, B Li, X Ding, A Hussain… - Advanced …, 2020 - Wiley Online Library
Life sciences have been revolutionized by genome editing (GE) tools, including zinc finger
nucleases, transcription activator‐Like effector nucleases, and CRISPR (clustered regulatory …

Human genetic diversity alters off-target outcomes of therapeutic gene editing

S Cancellieri, J Zeng, LY Lin, M Tognon, MA Nguyen… - Nature …, 2023 - nature.com
CRISPR gene editing holds great promise to modify DNA sequences in somatic cells to treat
disease. However, standard computational and biochemical methods to predict off-target …

A new age of precision gene therapy

A Schambach, CJ Buchholz, R Torres-Ruiz, K Cichutek… - The Lancet, 2024 - thelancet.com
Gene therapy has become a clinical reality as market-approved advanced therapy medicinal
products for the treatment of distinct monogenetic diseases and B-cell malignancies. This …

In vivo CRISPR editing with no detectable genome-wide off-target mutations

P Akcakaya, ML Bobbin, JA Guo, J Malagon-Lopez… - Nature, 2018 - nature.com
CRISPR–Cas genome-editing nucleases hold substantial promise for develo** human
therapeutic applications,,,,–but identifying unwanted off-target mutations is important for …

CHANGE-seq reveals genetic and epigenetic effects on CRISPR–Cas9 genome-wide activity

CR Lazzarotto, NL Malinin, Y Li, R Zhang, Y Yang… - Nature …, 2020 - nature.com
Current methods can illuminate the genome-wide activity of CRISPR–Cas9 nucleases, but
are not easily scalable to the throughput needed to fully understand the principles that …

Whole genome sequencing reveals rare off‐target mutations and considerable inherent genetic or/and somaclonal variations in CRISPR/Cas9‐edited cotton plants

J Li, H Manghwar, L Sun, P Wang… - Plant biotechnology …, 2019 - Wiley Online Library
The CRISPR/Cas9 system has been extensively applied for crop improvement. However,
our understanding of Cas9 specificity is very limited in Cas9‐edited plants. To identify on …

Evaluating and enhancing target specificity of gene-editing nucleases and deaminases

D Kim, K Luk, SA Wolfe, JS Kim - Annual review of biochemistry, 2019 - annualreviews.org
Programmable nucleases and deaminases, which include zinc-finger nucleases,
transcription activator-like effector nucleases, CRISPR RNA-guided nucleases, and RNA …

Genome-wide detection of CRISPR editing in vivo using GUIDE-tag

SQ Liang, P Liu, JL Smith, E Mintzer, S Maitland… - Nature …, 2022 - nature.com
Abstract Analysis of off-target editing is an important aspect of the development of safe
nuclease-based genome editing therapeutics. in vivo assessment of nuclease off-target …

The current state and future of CRISPR-Cas9 gRNA design tools

LOW Wilson, AR O'Brien, DC Bauer - Frontiers in pharmacology, 2018 - frontiersin.org
Recent years have seen the development of computational tools to assist researchers in
performing CRISPR-Cas9 experiment optimally. More specifically, these tools aim to …