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Results and Publications

Peer-reviewed publications

 Thumann et al. Engineering of PEDF-Expressing Primary Pigment Epithelial Cells by the SB Transposon System Delivered by pFAR4 Plasmids. Mol Ther Nucleic Acids. 2017. 6:302-314.

Hudecek et al. Going non-viral: the Sleeping Beauty transposon system breaks on through to the clinical side. Crit Rev Biochem Mol Biol. 2017. 12:1-26.

Stolba et al. Neovascular AMD in Austria – an expert review – and introduction to the TargetAMD approach. Spektrum der Augenheilkunde. Resubmitted after “accepted with minor comments”.

Fernandez-Robredo et al. Long-term PEDF release in rat iris and retinal epithelial cells after Sleeping Beauty transposon-mediated gene delivery. Mol Ther Nucleic Acids. Under revision

Pastor et al. The antibiotic-free pFAR4 vector merged with the Sleeping Beauty transposon system mediates efficient transgene delivery in human cells. In preparation.

Citable abstracts

Citable abstracts 2012-2017

Dissemination activities

Partner Act. Title Date Place Audience
UNIGE PRE Séance publique d’information aux patients 2012-12-06 2013-03-14 2013-05-23 2013-11-21 2014-01-16 Geneva (CH) Patients
UNIGE PRE Journée de la rétine (La thérapie génique: espoir ou illusion?) 2012-03-21 Coppet (CH) Clinicians
UNIGE COLL
PRE
Neuroclinique colloquium (Non-viral Gene-therapeutic Approach to Treat AMD – The TargetAMD Project) 2012-04-17 Geneva (CH) Neuro- logists
CNRS CONF
PRE
ESGCT-SFTCG Conference 2012-10-25 Paris (FR) Scientific Community
UNIGE COLL
PRE
Internists colloquium (Dégénérence Maculaire Liée à l’Âge (DMLA)) 2012-09-24 Geneva (CH) Internists
UKA PRE Electrochemotherapy, 2nd International Users’ Meeting 2013-03-01 -2013-03-02 Bologna (I) Scientific Community
UNIGE LECT
PRE
Pathologies vitréorétinienne 2013 Geneva (CH) Students
UNIGE TRAI
PRE
Colloquium for nurses 2013-04-18 Geneva (CH) Nurses
UNIGE CONF
PRE
DOC (Cell-based Therapy for the Treatment of Retinal Degeneration) 2013-05-15 -2013-05-17 Nurem- berg (GER) Scientific community
CNRS CONF
PRE
ASGCT – American Society of Cell and Gene Therapy 2013-05-18 Salt Lake City (US) Scientific Community
UNIGE PRE Leçon inaugurale (La thérapie génique: espoir ou illusion?) 2013-05-23 Geneva (CH) Employees of the UNIGE
UNIGE ARTI Pulsations (Espoir pour la DMLA) 2013-05-01 Geneva (CH) Public
UNIGE CONF
PRE
SOE (Cell-based Therapy for the Treatment of Retinal Degeneration) 2013-06-08 -2013-06-11 Copen- hagen (DK) Scientific community
KAR PRE SOE, Retina showcase (Human retinal pigment epithelial transplantation – are we too slow? 2013-06-08 -2013-06-11 Copen- hagen (DK) Scientific community
KAR PRE Deutsche Ophthalmochirurgen; Symposium on cell therapies: Human RPE Transplantation- are we too slow? 2013-06-12 -2013-06-14 Nuremberg (GER) Scientific community
UKA PRE 25. Aachener Dialog, “Hinterabschnittserkrankungen” 2013-06-19 Aachen (GER) Ophthal- mologists
KAR PRE Deutsche Ophthalmologische Gesellschaft 2013-09-16 Berlin (GER) Ophthal- mologists
UKA CONF
PRE
POS
Conference on Transposition and Genome Engineering 2013-09-18 Budapest (HUN) Scientific Community
MDC PEI CONF
POS
Conference on Transposition and Genome Engineering: (Transposon-Mediated PEDF Gene Delivery into Primary Pigment Epithelial Cells for the Treatment of Retinal Degenerative Diseases) 2013-09-18 -2013-09-21 Budapest (HUN) Scientific community
UNIGE PRE Age-related Macular Degeneration 2013-10-17 Geneva (CH) Employees of the UNO
UKA PRE
POS
European Society of Gene & Cell Therapy 2013-10-25 -2013-10-28 Madrid (ESP) Scientific Community
UNIGE CONF
POS
ESGCT and SETGyC collaborative congress 2013-10-25 -2013-10-28 Madrid (ES) Scientific Community
UKA CONF
POS
ESGCT and SETGyC collaborative congress 2013-10-25 -2013-10-28 Madrid (ES) Scientific Community
CNRS POS Conference on Transposition and Genome Engineering – 2013 2013-09-18 -2013-09-21 Budapest (HUN) Scientific Community
CNRS POS European Society of Gene & Cell Therapy Conference -2013 2013-10-25 -2013-10-28 Madrid (ES) Scientific Community
CNRS CONF
PRE
BIT’s 4th Worlg Gene Convention) 2013-11-13 Haikou (CHN) Scientific Community
CNRS CONF
LECT
Lecture to students – Master of Gene and Cell Therapy 2013-11-07 Paris XII University (FR) Students
CNRS CONF Lecture to students on Biomedicines 2013-11-26 Paris (FR) Students
CNRS WS Workshop on Gene Therapy 2013-11-26 Doha (QA) Scientific Community
CNRS SEM Hadassah Medical School 2013-12-02 Jerusalem (ISR) Scientific Community
CNRS SEM Weizmann Institute 2013-12-04 Rehovot (ISR) Scientific Community
CNRS CONF Lecture to students – Master of Biotherapies 2014-01-13 Paris XI University (FR) Scientific Community
CNRS LECT Lecture to Master students in Biomedicine 2014-01-28 – ESPCI- ParisTech (FR) Students
CNRS CONF E-rare Symposium 2014-01-21 Athens (GRC) Scientific Community
CNRS CONF History of plasmid size reduction Plenary Introductory lecture – Minicircle & DNA Vector Conference – 2014 2014-01-28 Bielefeld (GER) Scientific Community
UNIGE POS Société Française de Thérapie Cellulaire et Génique 2014-03-19 Toulouse (Fr) Scientific Community
UNIGE CONF Retina 2014 2014-03-29 Rome (I) Scientific Community
CNRS CONF Minicircle & DNA Vector Conference – 2014 2014-05-07 -2014-05-09 Bielefeld (GER) Scientific Community
UKA POS
PRE
Association for Research in Vision and Ophthalmology 2014-05-04 -2014-05-08 Orlando, Florida (US) Scientific Community
UNIGE CONF Jules Gonin 2014 2014-05-09 Zurich (CH) Scientific Community
CNRS POS American Society of Gene & Cell Therapy Conference – 2014 2014-05-21 -2014-05-24 Washing- ton (US) Scientific Community
GenoS WEB Presentation of the Project in the GenoSafe new website June 2014 Web Public
UNIGE SEM Lecture to ophthalmologists 2014-09-09 Geneva (CH) Ophthal- mologists
CNRS LECT Université Paris-Descartes Biotherapy 2014-09-28 Paris (FR) Students
UN CONF EVER 2014. European association for vision and eye research 2014-10-01 -2014-10-04 Nice (I) Scientific community
UNIGE POS ESGCT – NVGC Joint meeting The Hague 2014-10-22 -2014-10-26 The Hague (NL) Scientific Community
GenoS NEWL Presentation of the Project in the GenoSafe Newsletter 2014-10-20 Scientific Community
UN CONF Use of Antibiotic-Resistance-Free Plasmids for Non-Viral Transfection of Pigment Epithelium-Derived Factor in Rat Primary Cells. 2014 Nice
(FR)
Scientific Community
UNIGE PRE Diabetic Retinopathy 2014-11-13 Geneva (CH) Employees of the UNO
UNIGE PRE IFA – Innsbrucker Fortbildung für Augenärzte: Gene-Therapy instead of Avastin 2014-11-15 Insbruck (AUS) Scientific Community
UNIGE PRE Ex vivo Gene Therapy for the Treatment of exudative Age-Related Macular Degeneration (Retina Now – Novartis Symposium) 2015-03-05 Berne (CH) Scientific Community
UNIGE PUB Gentherapie statt Anti-VEGF bei AMD (Ophthalmologische Nachrichten, Apr 2015) 2015-04-01 Germany Scientific Community
UKA POS Analysis of Potential Tumorigenicity of PEDF-Transfected Primary Human Retinal Pigment Epithelial Cells Using Soft Agar Assay (Association for Research in Vision and Ophthalmology – ARVO 2015) 2015-05-04 Denver (USA) Scientific Community
UN POS Biodistribution of Rat Primary Cells transfected with SB100X transposon-mediated Pigment Epithelium-Derived Factor (PEDF) gene in Brown Norway rats after subretinal injection (ARVO 2015) 2015-05-03 Denver (USA) Scientific Community
UNIGE POS The use of mini-plasmids free of antibiotic resistance markers for a gene therapeutical approach to treat AMD (ARVO 2015) 2015-05-03 Denver (USA) Scientific Community
UNIGE POS pFAR4 miniplasmids in combination with the Sleeping Beauty transposon system allow efficient transfection of freshly isolated iris pigment epithelial cells (ARVO 2015) 2015-05-03 Denver (USA) Scientific Community
UNIGE POS Transposon-Based, Targeted Ex Vivo Gene Therapy to Treat Age-Related Macular Degeneration (ARVO 2015) 2015-05-03 Denver (USA) Scientific Community
UNIGE PRE Gene therapy in ophthalmology – the TargetAMD project (TEDD Symposium – Frontiers in Personalized Medicine) 2015-06-18 Wädenswil (CH) Scientific Community
UNIGE PUB Gene therapy for the eye: the TargetAMD project (CHIMIA 2015, 69, No. 7/8, 491-493) 2015-08-17 Berne (CH) Scientific Community
CNRS PRES 1st World Congress on Electroporation 2015-09-06 Portoroz, Slovenia Scientific Community
UKA POS pFAR4/SB100X-mediated PEDF gene delivery in primary murine pigment epithelial cells (European Society of Gene & Cell Therapy – ESGCT 2015) 2015-09-17 Helsinki (FN) Scientific Community
UNIGE POS Over-expression of PEDF by PEDF-transfected primary pigment epithelial cells does not induce tumorigenicity (ESGCT 2015) 2015-09-17 Helsinki (FN) Scientific Community
UNIGE POS Stable genomic integration of PEDF in primary pigment epithelial cells transfected with the Sleeping Beauty transposon system to treat age-related macular degeneration (AMD)(ESGCT 2015) 2015-09-17 Helsinki (FN) Scientific Community
UNIGE PRE The TargetAMD project – Using Free of antibiotic resistance gene (pFAR4) miniplasmids for a Sleeping Beauty (SB100X) mediated gene therapy to treat neovascular Age-Related Macular Degeneration (nAMD) (ESGCT 2015) 2015-09-17 Helsinki (FN) Scientific Community
UNIGE
RWTH
UKA
POS Information poster TargetAMD (firstly presented: ESGCT 2015) 2015-09-17 Helsinki (FN) Scientific Community
UNIGE
RWTH
UKA
FLY Information flyer TargetAMD (firstly presented: ESGCT 2015) 2015-09-17 Helsinki (FN) Scientific Community
UKA POS Lack of Tumorigenic Potential of PEDF Transfected Primary Human Pigment Epithelial Cells in Soft Agar Assays 2015-10-04-01 Berlin (GER) Scientific Community
UNIGE ART Gentherapie zur Behandlung der nAMD: das TargetAMD Projekt 2016-01 (GER) Scientific Community
UNIGE LECT Transposon-based ex vivo gene therapy to treat age-related macular degeneration 2016-01-21 Neuchâtel (CH) Scientific Community
UKA LECT Nicht-virale ex vivo Gentherapie zur Behandlung der neovaskulären altersbedingten Makuladegeneration (178. Versammlung des Vereins Rheinisch-Westfälischer Augenärzte – RWA 2016) 2016-01-30 Bonn(GER) Scientific Community
UNIGE LECT Status quo of TargetAMD_RPE at KAR (GM TargetAMD Consortium) 2016-04-22 (CH) Scientific Community
KAR LECT Presentation of TargetAMD_RPE to ophthalmologists and optometrists (Eye Dept., KAR) 2016-04-28 (AUS) Scientific Community
UN POS Efficacy of Rat Primary Cells transfected with Pigment Epithelium-Derived Factor using the Sleeping BeautyTransposon system in choroidal neovascularization (ARVO 2016) 2016-04-30 Seattle (USA) Scientific Community
UNIGE POS Results of a biodistribution study of Venus transfected pigment epithelial cells transplanted subretinally in rabbits (ARVO 2016) 2016-04-30 Seattle (USA) Scientific Community
UNIGE POS Human PEDF optimized gene for transposon-based gene therapy to treat age-related macular degeneration (ARVO 2016) 2016-04-30 Seattle (USA) Scientific Community
UNIGE POS Optimized Non-Viral Transfection of human RPE and IPE cells used for a Gene-Therapeutic Treatment of neovascular AMD (ARVO 2016) 2016-04-30 Seattle (USA) Scientific Community
KAR LECT Presentation of TargetAMD_RPE to Pharmacists 2016-05-03 (AUS) Scientific Community
PEI LECT The Sleeping Beauty Transposon System for Molecular Therapy (Annual Congress of the American Society of Gene and Cell Therapy, ASGCT) 2016-05-04 Washington (USA) Scientific Community
UNIGE LECT La dégénérescence maculaire liée à l’âge (DMLA) – ses causes et les possibilités de traitement (patient information event) 2016-05-12 Geneva (CH) Scientific Community
UKA LECT Pigment Epithelial Cell-Based Non-Viral Gene Therapeutic Treatment of Neovascular AMD (Cellular and Molecular Neurobiology Study Group Meeting, Uniklinik RWTH Aachen) 2016-05-30 Aachen(GER) Scientific Community
KAR LECT Presentation of TargetAMD_RPE to Laboratory Employees 2016-06-02 (AUS) Scientific Community
UKA LECT TargetAMD Transposon-based, targeted ex vivo gene therapy to treat age-related macular degeneration (Seminar der Klinik für Augenheilkunde, Uniklinik RWTH Aachen) 2016-06-22 Aachen (GER) Scientific Community
UKA LECT Improved pigment epithelial cell-based non-viral gene therapeutic approach for neovascular AMD (Deutsche Ophthalmologische Gesellschaft – DOG Kongress 2016) 2016-06-29 Berlin(GER) Scientific Community
UKA POS Lack of indication for tumorigenicity after pFAR4/SB100X-mediated PEDF gene delivery in primary murine pigment epithelial cells ex vivo (European Society of Gene & Cell Therapy – ESGCT 2016) 2016-10-09 & 10 Florence (I) Scientific Community
UKA POS pFAR4/SB100X-mediated PEDF gene delivery in iris pigment epithelial cells isolated from patients’ iridectomies mimicking a therapeutic approach for the treatment of neovascular AMD (European Society of Gene & Cell Therapy – ESGCT 2016) 2016-10-09 & 10 Florence (I) Scientific Community
UKA POS pFAR4/SB100X-mediated PEDF gene delivery in primary human pigment epithelial cells for the treatment of neovascular AMD (European Society of Gene & Cell Therapy – ESGCT 2016) 2016-10-09 & 10 Florence (I) Scientific Community
UNIGE POS Safety Studies for Cell-Based Gene Therapy (TargetAMD project): In Vivo Exclusion of Tumorigenicity and Proof of Cell Product Quality (European Society of Gene & Cell Therapy – ESGCT 2016) 2016-10-09 & 10 Florence (I) Scientific Community
UNIGE POS Transposon-Based, Targeted Ex Vivo Gene Therapy to Treat Age-related Macular Degeneration: The TargetAMD projectEuropean Society of Gene & Cell Therapy – ESGCT 2016) 2016-10-09 & 10 Florence (I) Scientific Community (European Society of Gene & Cell Therapy – ESGCT 2016)
UNIGE POS Translation of GLP-grade Electroporation of Primary Pigment Epithelial Cells to GMP-grade GTMP Manufacturing for Clinical Use (European Society of Gene & Cell Therapy – ESGCT 2016) 2016-10-09 & 10 Florence (I) Scientific Community
Activities (Act.)
ARTI Artikel in press
COLL Colloquium
CONF Conference
LECT Lecture
NEWL Newsletter
POS Poster
PRE Presentation
SEM Seminar
TRAI Training
WEB Website
WS Workshop