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List of works by Gwladys Gernoux

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

scientific article published on 10 April 2017

A divalent siRNA chemical scaffold for potent and sustained modulation of gene expression throughout the central nervous system

scientific article published on 02 August 2019

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

Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study

scientific article published on 02 February 2019

CHOP and c-JUN upregulate the mutant Z alpha-1 antitrypsin, exacerbating its aggregation and liver proteotoxicity

scientific article published on 28 July 2020

Early interaction of adeno-associated virus serotype 8 vector with the host immune system following intramuscular delivery results in weak but detectable lymphocyte and dendritic cell transduction

scientific article

Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity

scientific article published on 16 April 2019

Liver-specific transcriptional modules identified by genome-wide in silico analysis enable efficient gene therapy in mice and non-human primates

scientific article

Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping ⬇️

scientific article published on March 10, 2011

Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

scientific article published on 13 January 2020

Regulatory and Exhausted T Cell Responses to AAV Capsid.

scientific article published on 21 March 2017

SOD1 Suppression with Adeno-Associated Virus and MicroRNA in Familial ALS

scientific article published on 01 July 2020

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

scientific article published on 29 December 2015

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