List of works - Florie Borel

5 Year Expression and Neutrophil Defect Repair after Gene Therapy in Alpha-1 Antitrypsin Deficiency.

scientific article published on 10 April 2017

Adenosine triphosphate-binding cassette transporter genes up-regulation in untreated hepatocellular carcinoma is mediated by cellular microRNAs.

scientific article published on 13 January 2012

Apolipoprotein B knockdown by AAV-delivered shRNA lowers plasma cholesterol in mice

scientific article published on 8 February 2011

Artificial miRNAs reduce human mutant Huntingtin throughout the striatum in a transgenic sheep model of Huntington's disease.

scientific article published on 5 December 2017

Design of AAV Vectors for Delivery of RNAi

scientific article published on 01 January 2019

Design, Cloning, and In Vitro Screening of Artificial miRNAs to Silence Alpha-1 Antitrypsin.

scientific article published in January 2017

Diagnostic and therapeutic potential of miRNA signatures in patients with hepatocellular carcinoma

scientific article

Editing out five Serpina1 paralogs to create a mouse model of genetic emphysema

scientific article published on 16 February 2018

Embedding siRNA sequences targeting apolipoprotein B100 in shRNA and miRNA scaffolds results in differential processing and in vivo efficacy

scientific article published on 23 October 2012

In vivo knock-down of multidrug resistance transporters ABCC1 and ABCC2 by AAV-delivered shRNAs and by artificial miRNAs.

scientific article published on 17 June 2011

Intrathecal Delivery of AAV Vectors in Cynomolgus Macaques for CNS Gene Therapy and Gene Expression Analysis in Microdissected Motor Neurons

scientific article published on 01 January 2019

Optimization and comparison of knockdown efficacy between polymerase II expressed shRNA and artificial miRNA targeting luciferase and Apolipoprotein B100.

scientific article

Quantification of Z-AAT by a Z-Specific "Sandwich" ELISA.

scientific article published in January 2017

Recombinant AAV as a platform for translating the therapeutic potential of RNA interference.

scientific article published on 19 December 2013

Safe and Efficient Silencing with a Pol II, but Not a Pol lII, Promoter Expressing an Artificial miRNA Targeting Human Huntingtin.

scientific article published on 14 April 2017

Safe and effective superoxide dismutase 1 silencing using artificial microRNA in macaques

scientific article published on 01 October 2018

Survival Advantage of Both Human Hepatocyte Xenografts and Genome-Edited Hepatocytes for Treatment of α-1 Antitrypsin Deficiency.

scientific article published on 25 September 2017

Therapeutic expression of hairpins targeting apolipoprotein B100 induces phenotypic and transcriptome changes in murine liver

scientific article published on 24 October 2013

Therapeutic rAAVrh10 Mediated SOD1 Silencing in Adult SOD1(G93A) Mice and Nonhuman Primates.

scientific article published on 29 December 2015

List of works by Florie Borel