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European Network for the advancement of Clinical Gene Transfer & Therapy : EC-NoE fostering interaction of all stakeholders in the field in order to facilitate and help harmonise Ethical, Quality, Efficacy and Regulatory Issues.

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Publications from CliniGene partners

Partner Publications
Yla-Herttuala In vivo SPECT/CT imaging of human orthotopic ovarian carcinoma xenografts with 111In-labeled monoclonal antibodies.
Plasmid Factory Transgene expression of transfected supercoiled plasmid DNA concatemers in mammalian cells.
Genosafe Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
Yla-Herttuala Eight-year safety follow-up of coronary artery disease patients after local intracoronary VEGF gene transfer.
Aiuti Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: analysis of function and distribution in lymphoid organs.
Aiuti New insights into the pathogenesis of adenosine deaminase-severe combined immunodeficiency and progress in gene therapy.
Aiuti Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.
Aiuti Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome.
Aiuti Ten years of gene therapy for primary immune deficiencies.
Aiuti Comprehensive genomic access to vector integration in clinical gene therapy.
Cartier Hematopoietic stem cell transplantation and hematopoietic stem cell gene therapy in X-linked adrenoleukodystrophy.
Scherman Generation of high-titer neutralizing antibodies against botulinum toxins A, B, and E by DNA electrotransfer.
Scherman Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery.
Scherman A comparison of synthetic oligodeoxynucleotides, DNA fragments and AAV-1 for targeted episomal and chromosomal gene repair.
Scherman Effects of ciliary muscle plasmid electrotransfer of TNF-alpha soluble receptor variants in experimental uveitis.
Scherman Skin-specific promoters for genetic immunisation by DNA electroporation.
Scherman Amphiphilic perfluoroalkyl carbohydrates as new tools for liver imaging.
Scherman Iminothiol/thiourea tautomeric equilibrium in thiourea lipids impacts DNA compaction by inducing a cationic nucleation for complex assembly.
Scherman Evaluation of the muscle gene transfer activity of a series of amphiphilic triblock copolymers.
Scherman Comparative gene transfer between cationic and thiourea lipoplexes.
Scherman Advantages of bioluminescence imaging to follow siRNA or chemotherapeutic treatments in osteosarcoma preclinical models.
Scherman pFARs, plasmids free of antibiotic resistance markers, display high-level transgene expression in muscle, skin and tumour cells.
Scherman Lipopolythiourea/DNA interaction: a biophysical study.
Scherman Nucleic acid transfer with hemifluorinated polycationic lipids.
Scherman Design and evaluation of histidine-rich amphipathic peptides for siRNA delivery.
Panet Thermosensitivity of the reverse transcription process as an inactivation mechanism of lentiviral vectors.
Panet The oncolytic activity of Newcastle disease virus NDV-HUJ on chemoresistant primary melanoma cells is dependent on the proapoptotic activity of the inhibitor of apoptosis protein Livin.
Panet Herpes simplex virus delivery to orthotopic rectal carcinoma results in an efficient and selective antitumor effect.
Mir Nucleic acids electrotransfer-based gene therapy (electrogenetherapy): past, current, and future.
Mir Electroporating fields target oxidatively damaged areas in the cell membrane.
Mermod Protein-binding microarrays: probing disease markers at the interface of proteomics and genomics.
Mermod Transcription factor CTF1 acts as a chromatin domain boundary that shields human telomeric genes from silencing.
Mermod MAR elements regulate the probability of epigenetic switching between active and inactive gene expression.
Mermod Gene-mediated restoration of normal myofiber elasticity in dystrophic muscles.
Merten Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.
Merten Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors.
Kochanek Adenovirus Vector Production and Purification.
Klatzmann High diversity of the immune repertoire in humanized NOD.SCID.gamma c-/- mice.
von Kalle Christof Sleeping beauty transposition from nonintegrating lentivirus.
von Kalle Christof The inherent differentiation program of short-term hematopoietic repopulating cells changes during human ontogeny.
von Kalle Christof Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy.
von Kalle Christof Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease.
von Kalle Christof Retroviral vector performance in defined chromosomal Loci of modular packaging cell lines.
von Kalle Christof Hematopoietic activity of human short-term repopulating cells in mobilized peripheral blood cell transplants is restricted to the first 5 months after transplantation.
Jacobs Andreas Patient-tailored, imaging-guided, long-term temozolomide chemotherapy in patients with glioblastoma.
Jacobs Andreas Molecular imaging of gliomas.
Jacobs Andreas Methods to monitor gene therapy with molecular imaging.
Hauser Hansjörg Generation of anti-TLR2 intrabody mediating inhibition of macrophage surface TLR2 expression and TLR2-driven cell activation.
Hauser Hansjörg ZrO(HPO(4))(1-x)(FMN)(x): quick and easy synthesis of a nanoscale luminescent biomarker.
Hauser Hansjörg Efficient generation of clinical-grade genetically modified dendritic cells for presentation of multiple tumor-associated proteins.
Hauser Hansjörg Quality of Cell Products: Authenticity, Identity, Genomic Stability and Status of Differentiation.
Hauser Hansjörg Timing and targeting of cell-based VEGF165 gene expression in ischemic tissue.
Hauser Hansjörg Bone formation in trabecular bone cell seeded scaffolds used for reconstruction of the rat mandible.
Hauser Hansjörg Effect of seeding technique and scaffold material on bone formation in tissue-engineered constructs.
Hauser Hansjörg Retroviral vector performance in defined chromosomal Loci of modular packaging cell lines.
Hauser Hansjörg A new PG13-based packaging cell line for stable production of clinical-grade self-inactivating gamma-retroviral vectors using targeted integration.
Hauser Hansjörg Synthetic gene regulation circuits for control of cell expansion.
Hauser Hansjörg Recombinant protein expression by targeting pre-selected chromosomal loci.
Hauser Hansjörg Precise regulation of transgene expression level and control of cell physiology.
Hauser Hansjörg Timing and targeting of cell-based VEGF165 gene expression in ischemic tissue.
Gänsbacher In vivo analysis of retroviral gene transfer to chondrocytes within collagen scaffolds for the treatment of osteochondral defects.
Gänsbacher Impact of radiation therapy on the oncolytic adenovirus dl520: implications on the treatment of glioblastoma.
Gänsbacher Efficient and stable gene transfer of growth factors into chondrogenic cells and primary articular chondrocytes using a VSV.G pseudotyped retroviral vector.
Gänsbacher GDNF-transduced Schwann cell grafts enhance regeneration of erectile nerves.
Gänsbacher Good news on the clinical gene transfer front.
Gänsbacher Searching for the right timing of surgical delay: angiogenesis, vascular endothelial growth factor and perfusion changes in a skin-flap model.
Gänsbacher Non-viral VEGF(165) gene therapy--magnetofection of acoustically active magnetic lipospheres ('magnetobubbles') increases tissue survival in an oversized skin flap model.
Gänsbacher The influence of the stable expression of BMP2 in fibrin clots on the remodelling and repair of osteochondral defects.
Gänsbacher Physicobiochemical synergism through gene therapy and functional tissue engineering for in vitro chondrogenesis.
Gänsbacher Adenovirus-based virotherapy enabled by cellular YB-1 expression in vitro and in vivo.
Gänsbacher Combined reporter gene PET and iron oxide MRI for monitoring survival and localization of transplanted cells in the rat heart.
Gänsbacher Therapeutic vaccination with an interleukin-2-interferon-gamma-secreting allogeneic tumor vaccine in patients with progressive castration-resistant prostate cancer: a phase I/II trial.
Gänsbacher Tissue engineering of the anterior cruciate ligament-sodium dodecyl sulfate-acellularized and revitalized tendons are inferior to native tendons.
Gänsbacher Cancer gene therapy: present and future.
Gänsbacher Tissue inhibitor of metalloproteinases-1-induced scattered liver metastasis is mediated by host-derived urokinase-type plasminogen activator.
Gänsbacher Reporter gene PET for monitoring survival of transplanted endothelial progenitor cells in the rat heart after pretreatment with VEGF and atorvastatin.
Gänsbacher Tetracycline-regulated bone morphogenetic protein 2 gene expression in lentivirally transduced primary rabbit chondrocytes for treatment of cartilage defects.
Gahrton Gösta Natural killer cell-based immunotherapy in cancer: current insights and future prospects.
Gahrton Gösta Impact of chromosome 13 deletion and plasma cell load on long-term survival of patients with multiple myeloma undergoing autologous transplantation.
Gahrton Gösta Retroviral gene transfer into primary human natural killer cells.
Gahrton Gösta Safety analysis of ex vivo-expanded NK and NK-like T cells administered to cancer patients: a phase I clinical study.
Dickson George Lentiviral-mediated genetic correction of hematopoietic and mesenchymal progenitor cells from Fanconi anemia patients.
Dickson George Integration-deficient lentiviral vectors: a slow coming of age.
Dickson George Comprehensive genomic access to vector integration in clinical gene therapy.
Dickson George The COX-2 inhibitors, meloxicam and nimesulide, suppress neurogenesis in the adult mouse brain.
Dickson George The identification of chromosomal translocation, t(4;6)(q22;q15), in prostate cancer.
Dickson George Design of phosphorodiamidate morpholino oligomers (PMOs) for the induction of exon skipping of the human DMD gene.
Dickson George Adenovirus vector vaccination induces expansion of memory CD4 T cells with a mucosal homing phenotype that are readily susceptible to HIV-1.
Dickson George Efficient liver-directed gene transfer by in situ generation of retroviral vector from adenoviral templates.
Dickson George Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice.
Dickson George Gene therapy for muscular dystrophy: current progress and future prospects.
Dickson George Preliminary evaluation of a self-complementary AAV2/8 vector for hepatic gene transfer of human apoE3 to inhibit atherosclerotic lesion development in apoE-deficient mice.
Dickson George Molecular, cellular and physiological investigation of myostatin propeptide-mediated muscle growth in adult mice.
Carrondo Stabilization of gammaretroviral and lentiviral vectors: from production to gene transfer.
Carrondo Retroviral vector production under serum deprivation: The role of lipids.
Carrondo Thermosensitivity of the reverse transcription process as an inactivation mechanism of lentiviral vectors.
Carrondo Adenovirus Vector Production and Purification.
Cichutek Klaus Functional HIV-2- and SIVsmmPBj- derived lentiviral vectors generated by a novel polymerase chain reaction-based approach.
Cichutek Klaus Interaction of Vpx and apolipoprotein B mRNA-editing catalytic polypeptide 3 family member A (APOBEC3A) correlates with efficient lentivirus infection of monocytes.
Cichutek Klaus The US and EU regulatory perspectives on the clinical use of hematopoietic stem/progenitor cells genetically modified ex vivo by retroviral vectors
Cichutek Klaus Lentiviral vectors with measles virus glycoproteins - dream team for gene transfer?
Cichutek Klaus Restriction of HIV-1 replication in monocytes is abolished by Vpx of SIVsmmPBj.
Cichutek Klaus Pseudotyping lentiviral vectors with the wild-type measles virus glycoproteins improves titer and selectivity.
Bubenik Genetically modified vaccines augment the efficacy of cancer surgery and chemotherapy.
Bubenik Genetically modified cellular vaccines against human papillomavirus type 16 (HPV16)-associated tumors: adjuvant treatment of minimal residual disease after surgery/chemotherapy.
Bubenik HPV 16-associated tumours: IL-12 can repair the absence of cytotoxic and proliferative responses of tumour infiltrating cells after chemotherapy.
Bubenik Therapy for minimal residual tumor disease: beta-galactosylceramide inhibits the growth of recurrent HPV16-associated neoplasms after surgery and chemotherapy.
Bubenik Induction of protective immunity against MHC class I-deficient, HPV16-associated tumours with peptide and dendritic cell-based vaccines.
Bosch Deficiency of CB2 cannabinoid receptor in mice improves insulin sensitivity but increases food intake and obesity with age.
Bosch Endothelial cell transduction in primary cultures from regressing mesonephros.
Bosch Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material.
Bosch Production, Purification and Characterization of Adeno-Associated Vectors.
Bosch EuroPhenome: a repository for high-throughput mouse phenotyping data.
Bosch Hypothalamic-specific manipulation of Fto, the ortholog of the human obesity gene FTO, affects food intake in rats.
Bosch Skeletal muscle metabolism in the pathology and treatment of type 1 diabetes.
Bosch High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency.
Bosch Overexpression of kinase-negative protein kinase Cdelta in pancreatic beta-cells protects mice from diet-induced glucose intolerance and beta-cell dysfunction.
Bosch Scavenger function of resident autofluorescent perivascular macrophages and their contribution to the maintenance of the blood-retinal barrier.
Bosch Phosphofructo-1-kinase deficiency leads to a severe cardiac and hematological disorder in addition to skeletal muscle glycogenosis.
Bosch Gene and cellular therapy in Spain: moving forward.
Bosch Increased intraocular insulin-like growth factor-I triggers blood-retinal barrier breakdown.
Bosch Inducible adeno-associated virus vectors promote functional angiogenesis in adult organisms via regulated vascular endothelial growth factor expression.
Baum Retroviral vector performance in defined chromosomal Loci of modular packaging cell lines.
Baum A new PG13-based packaging cell line for stable production of clinical-grade self-inactivating gamma-retroviral vectors using targeted integration.
Baum Mechanisms controlling titer and expression of bidirectional lentiviral and gammaretroviral vectors.
Baum Limited complementarity between U1 snRNA and a retroviral 5' splice site permits its attenuation via RNA secondary structure.
Baum Gene therapy of MPL deficiency: challenging balance between leukemia and pancytopenia.
Baum Genotoxic potential of lineage-specific lentivirus vectors carrying the beta-globin locus control region.
Baum Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors.
Baum Cell-intrinsic and vector-related properties cooperate to determine the incidence and consequences of insertional mutagenesis.
Baum Protein Scaffold and Expression Level Determine Antiviral Activity of Membrane-Anchored Antiviral Peptides.
Baum Ectopic HOXB4 overcomes the inhibitory effect of tumor necrosis factor-{alpha} on Fanconi anemia hematopoietic stem and progenitor cells.
Baum Transcriptional enhancers induce insertional gene deregulation independently from the vector type and design.
Auricchio Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses.
Auricchio Abnormal autophagy, ubiquitination, inflammation and apoptosis are dependent upon lysosomal storage and are useful biomarkers of mucopolysaccharidosis VI.
Auricchio Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.
Auricchio Isolation and evaluation of novel adeno-associated virus sequences from porcine tissues.
Ali Robin Cell transplantation strategies for retinal repair.
Ali Robin Targeted disruption of outer limiting membrane junctional proteins (Crb1 and ZO-1) increases integration of transplanted photoreceptor precursors into the adult wild-type and degenerating retina.
Ali Robin Adult ciliary epithelial cells, previously identified as retinal stem cells with potential for retinal repair, fail to differentiate into new rod photoreceptors.
Ali Robin Comprehensive genomic access to vector integration in clinical gene therapy.
Ali Robin AAV-mediated knockdown of peripherin-2 in vivo using miRNA-based hairpins.
Ali Robin Gene therapy in the second eye of RPE65-deficient dogs improves retinal function.
Cohen Haguenauer Odile En route to ethical recommendations for gene transfer clinical trials.
Cohen Haguenauer Odile Opinion paper on the current status of the regulation of gene therapy in Europe.
Cohen Haguenauer Odile In vivo repopulation ability of genetically corrected bone marrow cells from Fanconi anemia patients.
Cohen Haguenauer Odile Comprehensive genomic access to vector integration in clinical gene therapy.
Cohen Haguenauer Odile A one-step prescreening for point mutations and large rearrangement in BRCA1 and BRCA2 genes using quantitative polymerase chain reaction and high-resolution melting curve analysis.
Cohen Haguenauer Odile Toward a Proportionate Regulatory Framework for Gene Transfer: A Patient Group-Led Initiative.
Cohen Haguenauer Odile Relevance of an Academic GMP Pan-European Vector Infra-structure (PEVI).
ALI Robin Bilateral adrenalectomy in treatment of disseminated breast cancer.
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