CRISPR/Cas9 genome editing in human hematopoietic stem cells

RO Bak, DP Dever, MH Porteus - Nature protocols, 2018 - nature.com
Genome editing via homologous recombination (HR)(gene targeting) in human
hematopoietic stem cells (HSCs) has the power to reveal gene–function relationships and …

Base Editors-Mediated Gene Therapy in Hematopoietic Stem Cells for Hematologic Diseases

C Zhang, J Xu, Y Wu, C Xu, P Xu - Stem Cell Reviews and Reports, 2024 - Springer
Base editors, developed from the CRISPR/Cas system, consist of components such as
deaminase and Cas variants. Since their emergence in 2016, the precision, efficiency, and …

Genome editing in stem cells for disease therapeutics

M Song, S Ramakrishna - Molecular biotechnology, 2018 - Springer
Programmable nucleases including zinc finger nucleases, transcription activator-like effector
nucleases, and clustered regularly interspaced short palindrome repeats (CRISPR)/CRISPR …

Applications of CRISPR-Cas9 for advancing precision medicine in oncology: from target discovery to disease modeling

M Ravichandran, D Maddalo - Frontiers in Genetics, 2023 - frontiersin.org
The clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-
associated protein 9 (Cas9)(CRISPR/Cas9) system is a powerful tool that enables precise …

CRISPR-derived genome editing therapies: Progress from bench to bedside

HA Rees, AC Minella, CA Burnett, AC Komor… - Molecular Therapy, 2021 - cell.com
The development of CRISPR-derived genome editing technologies has enabled the precise
manipulation of DNA sequences within the human genome. In this review, we discuss the …

Minimizing off-target mutagenesis risks caused by programmable nucleases

K Ishida, P Gee, A Hotta - International journal of molecular sciences, 2015 - mdpi.com
Programmable nucleases, such as zinc finger nucleases (ZFNs), transcription activator like
effector nucleases (TALENs), and clustered regularly interspersed short palindromic repeats …

CRISPR/Cas9-mediated gene editing. A promising strategy in hematological disorders

L Ugalde, S Fañanas, R Torres… - Cytotherapy, 2023 - Elsevier
The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system has
revolutionized the gene editing field, making it possible to interrupt, insert or replace a …

CRISPR‐Cas9: A cornerstone for the evolution of precision medicine

S Razzouk - Annals of Human Genetics, 2018 - Wiley Online Library
Modern genetic therapy incorporates genomic testing and genome editing. It is the finest
approach for precision medicine. Genome editing is a state‐of‐the‐art technology to …

Gene editing of hematopoietic stem cells: hopes and hurdles toward clinical translation

S Ferrari, V Vavassori, D Canarutto, A Jacob… - Frontiers in Genome …, 2021 - frontiersin.org
In the field of hematology, gene therapies based on integrating vectors have reached
outstanding results for a number of human diseases. With the advent of novel programmable …

CRISPR/Cas9‐mediated genome editing: From basic research to translational medicine

FV Jacinto, W Link, BI Ferreira - Journal of cellular and …, 2020 - Wiley Online Library
The recent development of the CRISPR/Cas9 system as an efficient and accessible
programmable genome‐editing tool has revolutionized basic science research …