PMID- 32203198
OWN - NLM
STAT- MEDLINE
DCOM- 20210818
LR  - 20240104
IS  - 1476-5462 (Electronic)
IS  - 0969-7128 (Linking)
VI  - 27
IP  - 9
DP  - 2020 Sep
TI  - 'Mini' U6 Pol III promoter exhibits nucleosome redundancy and supports 
      multiplexed coupling of CRISPR/Cas9 effects.
PG  - 451-458
LID - 10.1038/s41434-020-0142-z [doi]
AB  - RNA polymerase III (Pol III) promoters express short non-coding RNAs and have 
      been adopted for expression of microRNA, interference RNA, and CRISPR single 
      guide RNA (sgRNA). Vectors incorporating H1 and U6 Pol III promoters are being 
      applied for therapeutic genome editing, including multiplexed CRISPR/Cas9 
      effects. We report a nucleosome-depleted, minimal U6 promoter, which when 
      embedded within lentiviral long terminal repeat (LTR) regions, supports high 
      level transcriptional activity. Furthermore, duplex minimal H1 & U6 promoters 
      transcribed dual sgRNAs for simultaneous disruption of T cell receptor (TCR) and 
      human leukocyte antigen (HLA) molecules, supporting efficient generation of 
      'universal' CAR T cells.
FAU - Preece, Roland
AU  - Preece R
AD  - Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of 
      Child Health, London, WC1N 1EH, UK.
FAU - Georgiadis, Christos
AU  - Georgiadis C
AUID- ORCID: 0000-0003-0031-8396
AD  - Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of 
      Child Health, London, WC1N 1EH, UK. christos.georgiadis@ucl.ac.uk.
FAU - Gkazi, Soragia Athina
AU  - Gkazi SA
AD  - Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of 
      Child Health, London, WC1N 1EH, UK.
FAU - Etuk, Aniekan
AU  - Etuk A
AD  - Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of 
      Child Health, London, WC1N 1EH, UK.
FAU - Christi, Abraham
AU  - Christi A
AD  - Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of 
      Child Health, London, WC1N 1EH, UK.
FAU - Qasim, Waseem
AU  - Qasim W
AD  - Molecular and Cellular Immunology Unit, UCL Great Ormond Street Institute of 
      Child Health, London, WC1N 1EH, UK.
AD  - NIHR Great Ormond Street Hospital Biomedical Research Centre, 30 Guilford Street, 
      London, WC1N 1EH, UK.
LA  - eng
GR  - RP-2014-05-007/DH_/Department of Health/United Kingdom
GR  - IS-BRC-1215-20012/Great Ormond Street Hospital for Children (GOSH)/International
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20200320
PL  - England
TA  - Gene Ther
JT  - Gene therapy
JID - 9421525
RN  - 0 (Nucleosomes)
RN  - 0 (RNA, Guide, CRISPR-Cas Systems)
SB  - IM
MH  - *CRISPR-Cas Systems
MH  - Gene Editing
MH  - Humans
MH  - *Nucleosomes/genetics
MH  - Promoter Regions, Genetic
MH  - RNA, Guide, CRISPR-Cas Systems
EDAT- 2020/03/24 06:00
MHDA- 2021/08/19 06:00
CRDT- 2020/03/24 06:00
PHST- 2019/08/21 00:00 [received]
PHST- 2020/03/06 00:00 [accepted]
PHST- 2020/02/05 00:00 [revised]
PHST- 2020/03/24 06:00 [pubmed]
PHST- 2021/08/19 06:00 [medline]
PHST- 2020/03/24 06:00 [entrez]
AID - 10.1038/s41434-020-0142-z [pii]
AID - 10.1038/s41434-020-0142-z [doi]
PST - ppublish
SO  - Gene Ther. 2020 Sep;27(9):451-458. doi: 10.1038/s41434-020-0142-z. Epub 2020 Mar 
      20.