Imperial College London
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ProfessorNagyHabib

Faculty of Medicine, Department of Surgery & Cancer

Professor of Hepatobiliary Surgery
 
 
 
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Contact

 

+44 (0)20 3313 8574nagy.habib

 
 
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Assistant

 

Mrs Benita White +44 (0)7960 986 387

 
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Location

 

BN1/18 B BlockHammersmith HospitalHammersmith Campus

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Summary

 

Publications

Citation

BibTex format

@article{Setten:2018:10.2174/1389201019666180611093428,
author = {Setten, RL and Lightfoot, HL and Habib, NA and Rossi, JJ},
doi = {10.2174/1389201019666180611093428},
journal = {Current Pharmaceutical Biotechnology},
pages = {611--621},
title = {Development of MTL-CEBPA: Small Activating RNA Drug for Hepatocellular Carcinoma},
url = {http://dx.doi.org/10.2174/1389201019666180611093428},
volume = {19},
year = {2018}
}
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RIS format (EndNote, RefMan)

TY  - JOUR
AB  - BACKGROUND: Oligonucleotide drug development has revolutionised the drug discovery field allowing the notoriously "undruggable" genome to potentially become "druggable". Within this field, 'small' or 'short' activating RNAs (saRNA) are a more recently discovered category of short double stranded RNA with clinical potential. SaRNAs promote endogenous transcription from target loci, a phenomenon widely observed in mammals known as RNA activation (RNAa). The ability to target a particular gene is dependent on the sequence of the saRNA. Hence, the potential clinical application of saRNA is to increase target gene expression in a sequence specific manner. SaRNA based oligonucleotide therapeutics present great promise in expanding the "druggable" genome with particular areas of interest including transcription factor activation and haploinsufficency. Review and Conclusion: In this mini-review, we describe the pre-clinical development of the first saRNA drug to enter the clinic. This saRNA, referred to as MTL-CEBPA, induces transcription of the transcription factor CCAAT/enhancer-binding protein alpha (CEBPα), a tumour suppressor and critical regulator of hepatocyte function. MTL-CEBPA is presently in Phase I clinical trials for hepatocellular carcinoma (HCC). The clinical development of MTL-CEBPA will demonstrate "proof of concept", showing that saRNAs can provide the basis for drugs which enhance targeted gene expression and consequently improve disease outcome in patients.
AU  - Setten,RL
AU  - Lightfoot,HL
AU  - Habib,NA
AU  - Rossi,JJ
DO  - 10.2174/1389201019666180611093428
EP  - 621
PY  - 2018///
SN  - 1389-2010
SP  - 611
TI  - Development of MTL-CEBPA: Small Activating RNA Drug for Hepatocellular Carcinoma
T2  - Current Pharmaceutical Biotechnology
UR  - http://dx.doi.org/10.2174/1389201019666180611093428
UR  - https://www.ncbi.nlm.nih.gov/pubmed/29886828
UR  - http://hdl.handle.net/10044/1/60834
VL  - 19
ER  -
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