Romito, M;
Rai, R;
Thrasher, A;
Cavazza, A;
(2019)
Genome editing for blood disorders: state of the art and recent advances.
[Review].
Emerging Topics in Life Science
, 3
(3)
289 -299.
10.1042/ETLS20180147.
Preview |
Text
ETLS20180147.full.pdf - Published Version Download (1MB) | Preview |
Abstract
In recent years, tremendous advances have been made in the use of gene editing to precisely engineer the genome. This technology relies on the activity of a wide range of nuclease platforms — such as zinc-finger nucleases, transcription activator-like effector nucleases, and the CRISPR–Cas system — that can cleave and repair specific DNA regions, providing a unique and flexible tool to study gene function and correct disease-causing mutations. Preclinical studies using gene editing to tackle genetic and infectious diseases have highlighted the therapeutic potential of this technology. This review summarizes the progresses made towards the development of gene editing tools for the treatment of haematological disorders and the hurdles that need to be overcome to achieve clinical success.
| Type: | Article |
|---|---|
| Title: | Genome editing for blood disorders: state of the art and recent advances |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1042/ETLS20180147 |
| Publisher version: | https://doi.org/10.1042/ETLS20180147 |
| Language: | English |
| Additional information: | https://creativecommons.org/licenses/by/4.0/ This is an open access article published by Portland Press Limited on behalf of the Biochemical Society and the Royal Society of Biology and distributed under the Creative Commons Attribution License 4.0 (CC BY). |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Infection, Immunity and Inflammation Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10072094 |
Archive Staff Only
 |
View Item |
