CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside

O Humbert, C Samuelson… - British journal of …, 2021 - Wiley Online Library
Genome editing therapies represent a significant advancement in next‐generation,
precision medicine for the management of haematological diseases, and CRISPR/Cas9 has …

Therapeutic gene editing in haematological disorders with CRISPR/Cas9

TI Jensen, E Axelgaard, RO Bak - British journal of …, 2019 - Wiley Online Library
Summary The Clustered Regularly Interspaced Short Palindromic Repeats
(CRISPR)/CRISPR‐associated (Cas) 9 platform offers an efficient way of making precise …

[HTML][HTML] CRISPR to fix bad blood: a new tool in basic and clinical hematology

E González-Romero, C Martínez-Valiente… - …, 2019 - ncbi.nlm.nih.gov
Advances in genome engineering in the last decade, particularly in the development of
programmable nucleases, have made it possible to edit the genomes of most cell types …

CRISPR‐Cas9 technology and its application in haematological disorders

H Zhang, N McCarty - British journal of haematology, 2016 - Wiley Online Library
The recent advent of the Clustered Regularly Interspaced Short Palindromic Repeats
(CRISPR)‐CRISPR associated protein 9 (Cas9) system for precise genome editing has …

[HTML][HTML] A toolmaker's perspective on CRISPR-directed gene editing as a therapeutic strategy for leukemia and beyond

EB Kmiec, K Bloh - Expert Review of Hematology, 2021 - Taylor & Francis
We are molecular toolmakers. The tools we design, synthesize, and create disrupt or repair
the human genome. Either outcome can be achieved using bio-molecular scissors generally …

[HTML][HTML] CRISPR/Cas9 genome engineering in hematopoietic cells

D Sürün, H von Melchner, F Schnütgen - Drug Discovery Today …, 2018 - Elsevier
The development of genome editing tools capable of modifying specific genomic sequences
with unprecedented accuracy has opened up a wide range of new possibilities in targeted …

[HTML][HTML] Technical considerations for the use of CRISPR/Cas9 in hematology research

MC Gundry, DP Dever, D Yudovich, DE Bauer… - Experimental …, 2017 - Elsevier
Highlights•Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 is a
powerful tool for genome editing in hematopoietic cells, including primary hematopoietic …

[HTML][HTML] Utilization of CRISPR-mediated tools for studying functional genomics in hematological malignancies: an overview on the current perspectives, challenges …

M Solayappan, A Azlan, KZ Khor, NM Yusoff… - Frontiers in …, 2022 - frontiersin.org
Hematological malignancies (HM) are a group of neoplastic diseases that are usually
heterogenous in nature due to the complex underlying genetic aberrations in which …

[HTML][HTML] Utility of CRISPR/Cas9 systems in hematology research

D Lucas, HA O'Leary, BL Ebert, CA Cowan… - Experimental …, 2017 - Elsevier
Highlights•CRISPR/Cas9 overcomes limitations of earlier targeted nuclease-mediated
methods.•CRISPR/Cas9 allows for insertions/deletions of multiple alleles in a single …

[HTML][HTML] CRISPR medicine for blood disorders: Progress and challenges in delivery

T Mohammadian Gol, G Ureña-Bailén… - Frontiers in Genome …, 2023 - frontiersin.org
Blood disorders are group of diseases including hematological neoplasms, clotting
disorders and orphan immune deficiency diseases that affects human health. Current …