Publications
312 results found
Coutelle C, 2023, Comment on the the paper Persecutee in the Third Reich, full professor in the GDR: The communist physician Carl Coutelle (1908-1993) and his post-war career as pathologist by Nico Biermanns and Dominik Groβ. Pathol. Res. Pract. (2023) 246 154487, PATHOLOGY RESEARCH AND PRACTICE, Vol: 248, ISSN: 0344-0338
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- Citations: 1
Coutelle C, 2020, Gene Therapy for Cystic Fibrosis - Strategies, Problems and Perspectives, Concepts in Gene Therapy, Pages: 315-345, ISBN: 9783110808636
Al-Allaf FA, Abduljaleel Z, Athar M, et al., 2019, Modifying inter-cistronic sequence significantly enhances IRES dependent second gene expression in bicistronic vector: Construction of optimised cassette for gene therapy of familial hypercholesterolemia, NON-CODING RNA RESEARCH, Vol: 4, Pages: 1-14, ISSN: 2468-2160
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- Citations: 14
Coutelle C, 2018, An important step on the long path to clinical application of in utero gene therapy, GENE THERAPY, Vol: 25, Pages: 451-453, ISSN: 0969-7128
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- Citations: 1
Condiotti R, Goldenberg D, Giladi H, et al., 2014, Transduction of Fetal Mice With a Feline Lentiviral Vector Induces Liver Tumors Which Exhibit an E2F Activation Signature, MOLECULAR THERAPY, Vol: 22, Pages: 59-68, ISSN: 1525-0016
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- Citations: 11
Cheung W, Nowrouzi A, Coutelle C, et al., 2013, The fetal mouse is a model of genotoxicity that can uncover genes involved in liver cancer, Conference of the British-Society-for-Gene-and-Cell-Therapy (BSGCT), Publisher: MARY ANN LIEBERT INC, Pages: A15-A16, ISSN: 1043-0342
Nowrouzi A, Cheung WT, Li T, et al., 2013, The Fetal Mouse Is a Sensitive Genotoxicity Model That Exposes Lentiviral-associated Mutagenesis Resulting in Liver Oncogenesis, MOLECULAR THERAPY, Vol: 21, Pages: 324-337, ISSN: 1525-0016
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- Citations: 17
Argyros O, Wong S-P, Coutelle C, et al., 2013, Utilizing Minicircle Vectors for the Episomal Modification of Cells, MINICIRCLE AND MINIPLASMID DNA VECTORS: THE FUTURE OF NONVIRAL AND VIRAL GENE TRANSFER, Editors: Schleef, Publisher: BLACKWELL SCIENCE PUBL, Pages: 93-113, ISBN: 978-3-527-32456-9
Coutelle C, Waddington SN, 2012, Preface, Methods in Molecular Biology, Vol: 891, ISSN: 1064-3745
Coutelle C, Waddington SN, Themis M, 2012, Monitoring for potential adverse effects of prenatal gene therapy: mouse models for developmental aberrations and inadvertent germ line transmission., Methods Mol Biol, Vol: 891, Pages: 329-340
So far no systematic studies have been conducted to investigate developmental aberrations after prenatal gene transfer in mice. Here, we suggest procedures for such observations to be applied, tested and improved in further in utero gene therapy experiments. They are based on our own experience in husbandry for transgenic human diseases mouse models and breading, rearing, and observing mice after fetal gene transfer as well as on the systematic screens for monitoring of knock-out mutant mouse phenotypes established in international mutagenesis projects (EUMORPHIA and EUMODIC and subsequently the International Mouse Phenotyping Consortium). We also describe here the analysis procedures for detection of germ line mutations based on quantitative PCR (qPCR) by sperm-DNA analysis and breeding studies.
Coutelle C, Waddington SN, 2012, Vector systems for prenatal gene therapy: choosing vectors for different applications., Methods Mol Biol, Vol: 891, Pages: 41-53
This chapter gives a comparative review of the different vector systems applied to date in prenatal gene therapy experiments highlighting the need for versatility and choice for application in accordance with the actual aim of the study. It reviews the key characteristics of the four main gene therapy vector systems and gives examples for their successful application in prenatal gene therapy experiments.
Coutelle C, Waddington SN, 2012, The concept of prenatal gene therapy., Methods Mol Biol, Vol: 891, Pages: 1-7
This introductory chapter provides a short review of the ideas and practical approaches that have led to the present and perceived future development of prenatal gene therapy. It summarizes the advantages and the potential adverse effects of this novel preventive and therapeutic approach to the management of prenatal diseases. It also provides guidance to the range of conditions to which prenatal gene therapy may be applied and to the technical approaches, vectors, and societal/ethical considerations for this newly emerging field of Fetal Medicine.
Mehta V, Abi-Nader KN, Carr D, et al., 2012, Monitoring for potential adverse effects of prenatal gene therapy: use of large animal models with relevance to human application., Methods Mol Biol, Vol: 891, Pages: 291-328
Safety is an absolute prerequisite for introducing any new therapy, and the need to monitor the consequences of administration of both vector and transgene to the fetus is particularly important. The unique features of fetal development that make it an attractive target for gene therapy, such as its immature immune system and rapidly dividing populations of stem cells, also mean that small perturbations in pregnancy can have significant short- and long-term consequences. Certain features of the viral vectors used, the product of the delivered gene, and sometimes the invasive techniques necessary to deliver the construct to the fetus in utero have the potential to do harm. An important goal of prenatal gene therapy research is to develop clinically relevant techniques that could be applied to cure or ameliorate human disease in utero on large animal models such as sheep or nonhuman primates. Equally important is the use of these models to monitor for potential adverse effects of such interventions. These large animal models provide good representation of individual patient-based investigations. However, analyses that require defined genetic backgrounds, high throughput, defined variability and statistical analyses, e.g. for initial studies on teratogenic and oncogenic effects, are best performed on larger groups of small animals, in particular mice. This chapter gives an overview of the potential adverse effects in relation to prenatal gene therapy and describes the techniques that can be used experimentally in a large animal model to monitor the potential adverse consequences of prenatal gene therapy, with relevance to clinical application. The sheep model is particularly useful to allow serial monitoring of fetal growth and well-being after delivery of prenatal gene therapy. It is also amenable to serially sampling using minimally invasive and clinically relevant techniques such as ultrasound-guided blood sampling. For more invasive long-term monitoring, we describ
Coutelle C, Ashcroft R, 2012, Risks, benefits and ethical, legal, and societal considerations for translation of prenatal gene therapy to human application., Methods Mol Biol, Vol: 891, Pages: 371-387
The still experimental nature of prenatal gene therapy carries a certain degree of risk, both for the pregnant mother as well as for the fetus. Some of the risks are procedural hazards already known from more conventional fetal medicine interventions. Others are more specific to gene therapy such as the potential for interference with normal fetal development, the possibility of inadvertent germ line gene transfer, and the danger of oncogenesis. This chapter reviews the potential risks in relation to the expected benefits of prenatal gene therapy. It discusses the scientific, ethical, legal, and social implications of this novel preventive approach to genetic disease and outlines preconditions to be met in preparation for a potential future clinical application.
Bigger BW, Liao A-Y, Sergijenko A, et al., 2011, Trial and Error: How the Unclonable Human Mitochondrial Genome was Cloned in Yeast, PHARMACEUTICAL RESEARCH, Vol: 28, Pages: 2863-2870, ISSN: 0724-8741
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- Citations: 8
Conese M, Ascenzioni F, Boyd AC, et al., 2011, Gene and cell therapy for cystic fibrosis: From bench to bedside, JOURNAL OF CYSTIC FIBROSIS, Vol: 10, Pages: S114-S128, ISSN: 1569-1993
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- Citations: 48
Buckley SMK, Rahim AA, Chan JKY, et al., 2011, Recent advances in fetal gene therapy., Ther Deliv, Vol: 2, Pages: 461-469, ISSN: 2041-5990
Over the first decade of this new millennium gene therapy has demonstrated clear clinical benefits in several diseases for which conventional medicine offers no treatment. Clinical trials of gene therapy for single gene disorders have recruited predominantly young patients since older subjects may have suffered irrevocablepathological changes or may not be available because the disease is lethal relatively early in life. The concept of fetal gene therapy is an extension of this principle in that diseases in which irreversible changes occur at or beforebirth can be prevented by gene supplementation or repair in the fetus or associated maternal tissues. This article ccnsiders the enthusiasm and skepticism held for fetal gene therapy and its potential for clinical application. It coversa spectrum of candidate diseases for fetal gene therapy including Pompe disease, Gaucher disease, thalassemia, congenital protein C deficiency and cystic fibrosis. It outlines successful and not-so-successful examples of fetal gene therapy in animal models. Finally the application and potential of fetal gene transfer as a fundamental research tool for developmental biology and generation of somatic transgenic animals is surveyed.
Argyros O, Wong SP, Fedonidis C, et al., 2011, Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver, Journal of molecular medicine (Berlin, Germany), Vol: 89(5), Pages: 515-529
We have previously described the development of a scaffold/matrix attachment region (S/MAR) episomal vector system for in vivo application and demonstrated its utility to sustain transgene expression in the mouse liver for at least 6 months following a single administration. Subsequently, we observed that transgene expression is sustained for the lifetime of the animal. The level of expression, however, does drop appreciably over time. We hypothesised that by eliminating the bacterial components in our vectors, we could improve their performance since bacterial sequences have been shown to be responsible for the immunotoxicity of the vector and the silencing of its expression when applied in vivo. We describe here the development of a minimally sized S/MAR vector, which is devoid of extraneous bacterial sequences. This minicircle vector comprises an expression cassette and an S/MAR moiety, providing higher and more sustained transgene expression for several months in the absence of selection, both in vitro and in vivo. In contrast to the expression of our original S/MAR plasmid vector, the novel S/MAR minicircle vectors mediate increased transgene expression, which becomes sustained at about twice the levels observed immediately after administration. These promising results demonstrate the utility of minimally sized S/MAR vectors for persistent, atoxic gene expression.
Wong SP, Argyros O, Coutelle C, et al., 2011, Non-viral S/MAR vectors replicate episomally <i>in vivo</i> when provided with a selective advantage, GENE THERAPY, Vol: 18, Pages: 82-87, ISSN: 0969-7128
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- Citations: 34
Al-Allaf FA, Coutelle C, Waddington SN, et al., 2010, LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives., Int Arch Med, Vol: 3
Coronary artery diseases (CAD) inflict a heavy economical and social burden on most populations and contribute significantly to their morbidity and mortality rates. Low-density lipoprotein receptor (LDLR) associated familial hypercholesterolemia (FH) is the most frequent Mendelian disorder and is a major risk factor for the development of CAD. To date there is no cure for FH. The primary goal of clinical management is to control hypercholesterolaemia in order to decrease the risk of atherosclerosis and to prevent CAD. Permanent phenotypic correction with single administration of a gene therapeutic vector is a goal still needing to be achieved. The first ex vivo clinical trial of gene therapy in FH was conducted nearly 18 years ago. Patients who had inherited LDLR gene mutations were subjected to an aggressive surgical intervention involving partial hepatectomy to obtain the patient's own hepatocytes for ex vivo gene transfer with a replication deficient LDLR-retroviral vector. After successful re-infusion of transduced cells through a catheter placed in the inferior mesenteric vein at the time of liver resection, only low-level expression of the transferred LDLR gene was observed in the five patients enrolled in the trial. In contrast, full reversal of hypercholesterolaemia was later demonstrated in in vivo preclinical studies using LDLR-adenovirus mediated gene transfer. However, the high efficiency of cell division independent gene transfer by adenovirus vectors is limited by their short-term persistence due to episomal maintenance and the cytotoxicity of these highly immunogenic viruses. Novel long-term persisting vectors derived from adeno-associated viruses and lentiviruses, are now available and investigations are underway to determine their safety and efficiency in preparation for clinical application for a variety of diseases. Several novel non-viral based therapies have also been developed recently to lower LDL-C serum levels in FH patients. This article re
Rahim AA, Wong AMS, Buckley SMK, et al., 2010, <i>In utero</i> gene transfer to the mouse nervous system, BIOCHEMICAL SOCIETY TRANSACTIONS, Vol: 38, Pages: 1489-1493, ISSN: 0300-5127
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- Citations: 10
Wong S-P, Orestis A, Howe S, et al., 2010, Systemic Administration of Nonviral Vectors to Neonatal Mice, 7th Annual Meeting of German-Society-for-Gene-Therapy (DG-GT e.V.), Publisher: MARY ANN LIEBERT INC, Pages: 1198-1198, ISSN: 1043-0342
Argyros O, Wong S-P, Fedonidis C, et al., 2010, Development of Scaffold/Matrix Attachment Region(S/MAR) Minicircles for Enhanced and PersistentTransgene Expression in the Mouse Liver, 17th Annual Meeting of German Society for Gene Therapy (DG-GT e.V.), Publisher: Mary Ann Liebert Inc Publishers, Pages: 1197-1198
David AL, Abi-Nader KN, Weisz B, et al., 2010, Ultrasonographic development of the fetal sheep stomach and evaluation of early gestation ultrasound-guided in utero intragastric injection., Taiwan J Obstet Gynecol, Vol: 49, Pages: 23-29
OBJECTIVE: Safely targeting the fetal gastrointestinal tract during early gestation is essential to develop effective prenatal gene therapy for gastrointestinal diseases. In this study, we aimed to characterize the development of the fetal sheep stomach sonographically and to determine the optimum gestational age, as well as the shortterm morbidity and mortality of early-gestation ultrasound-guided intragastric injection. MATERIALS AND METHODS: In experiments investigating ultrasound-guided prenatal gene therapy, we studied the size and development of the stomach of 185 sheep fetuses (33-144 days' gestational age [GA]; term is 145 days). Ultrasound-guided intragastric injection was performed in 12 fetuses at 55-62 days' GA and postmortem examinations were performed 48 hours later. RESULTS: The stomach was not visible at or before 40 days' GA, but it was seen in all fetuses at 55 days' GA or more. The anteroposterior, transverse and longitudinal diameters of the stomach increased in a quasi-linear fashion throughout gestation. Intragastric injection was successful in 10 out of the 11 fetuses (91%) injected at 60-62 days' GA, with nine fetuses (91%) surviving this procedure. CONCLUSION: In the early-gestation sheep fetus, ultrasound-guided intragastric injection has a good success rate with a low short-term mortality and morbidity.
David AL, Abi-Nader KN, Weisz B, et al., 2010, ULTRASONOGRAPHIC DEVELOPMENT OF THE FETAL SHEEP STOMACH AND EVALUATION OF EARLY GESTATION ULTRASOUND-GUIDED <i>IN</i> <i>UTERO</i> I NTRAGASTRIC INJECTION, TAIWANESE JOURNAL OF OBSTETRICS & GYNECOLOGY, Vol: 49, Pages: 23-29, ISSN: 1028-4559
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- Citations: 3
Wong SP, Argyros O, Coutelle C, et al., 2009, Strategies for the episomal modification of cells, CURRENT OPINION IN MOLECULAR THERAPEUTICS, Vol: 11, Pages: 433-441, ISSN: 1464-8431
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- Citations: 14
Coutelle C, 2009, From mould to man Edward Lewis Tatum’s vision of the future of medicine, Cellular Therapy and Transplantation (CTT), Vol. 1, No. 4, 2009, Vol: 1
Wong SP, Argyros O, Coutelle C, et al., 2009, Development of S/MAR Minicircle Vectors for Persistent Expression <i>In Vivo</i>, 12th Annual Meeting of the American Society of Gene Therapy, Publisher: NATURE PUBLISHING GROUP, Pages: S286-S286, ISSN: 1525-0016
Al-Allaf FA, Mehmet H, Colakogullari M, et al., 2009, Tailoring the Size of Inter-Cistronic Spacer Sequence Significantly Increases IRES-Dependent Second Gene Expression over Cap-Dependent Expression in a Bicistronic Vector: Construction of Optimized 5'-hLDLR-IRES-eGFP-3' Cassette for Gene Therapy of Familial Hypercholesterolaemia, 12th Annual Meeting of the American Society of Gene Therapy, Publisher: NATURE PUBLISHING GROUP, Pages: S129-S130, ISSN: 1525-0016
Argyros O, Wong SP, Niceta M, et al., 2008, Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector, GENE THERAPY, Vol: 15, Pages: 1593-1605, ISSN: 0969-7128
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- Citations: 77
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