Publications
312 results found
Bigger BW, Siapati EK, Mistry A, et al., 2006, Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX, GENE THERAPY, Vol: 13, Pages: 117-126, ISSN: 0969-7128
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- Citations: 48
Coutelle C, Themis M, Waddington SN, et al., 2005, Gene therapy progress and prospects: Fetal gene therapy - first proofs of concept - some adverse effects, GENE THERAPY, Vol: 12, Pages: 1601-1607, ISSN: 0969-7128
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- Citations: 43
Themis M, Waddington SN, Schmidt M, et al., 2005, Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice, MOLECULAR THERAPY, Vol: 12, Pages: 763-771, ISSN: 1525-0016
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- Citations: 182
Buckley SMK, Waddington SN, Jezzard S, et al., 2005, Factors influencing adenovirus-mediated airway transduction in fetal mice, MOLECULAR THERAPY, Vol: 12, Pages: 484-492, ISSN: 1525-0016
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- Citations: 30
Weisz B, David AL, Gregory LG, et al., 2005, Targeting the respiratory muscles of fetal sheep for prenatal gene therapy for Duchenne muscular dystrophy, AMERICAN JOURNAL OF OBSTETRICS AND GYNECOLOGY, Vol: 193, Pages: 1105-1109, ISSN: 0002-9378
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- Citations: 12
Al-Allaf FA, Tolmachov O, Themis M, et al., 2005, Coupled analysis of bacterial transformants and ligation mixture by duplex PCR enables detection of fatal instability of a nascent recombinant plasmid, JOURNAL OF BIOCHEMICAL AND BIOPHYSICAL METHODS, Vol: 64, Pages: 142-146, ISSN: 0165-022X
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- Citations: 2
Waterhouse JE, Harbottle RP, Keller M, et al., 2005, Synthesis and application of integrin targeting lipopeptides in targeted gene delivery, CHEMBIOCHEM, Vol: 6, Pages: 1212-1223, ISSN: 1439-4227
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- Citations: 22
Waddington SN, Kramer MG, Hernandez-Alcoceba R, et al., 2005, <i>In utero</i> gene therapy:: Current challenges and perspectives, MOLECULAR THERAPY, Vol: 11, Pages: 661-676, ISSN: 1525-0016
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- Citations: 33
Al-Allaf F, Tolmachov O, Themis M, et al., 2005, Accurate size gauging of ExoIII/S1-generated deletions by PCR analysis of ligation mixtures, ANALYTICAL BIOCHEMISTRY, Vol: 339, Pages: 348-350, ISSN: 0003-2697
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- Citations: 2
Hoare J, Waddington S, Thomas HC, et al., 2005, Complement inhibition rescued mice allowing observation of transgene expression following intraportal delivery of baculovirus in mice, JOURNAL OF GENE MEDICINE, Vol: 7, Pages: 325-333, ISSN: 1099-498X
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- Citations: 38
Tolmachov O, Harbottle R, Bigger B, et al., 2005, Minimized, CpG-Depleted, and Methylated DNA Vectors: Toward Perfection in Nonviral Gene Therapy, DNA-Pharmaceuticals: Formulation and Delivery in Gene Therapy, DNA Vaccination and Immunotherapy, Editors: Schleef, Publisher: Wiley, Pages: 43-54, ISBN: 978-3-527-31187-3
Weisz B, David A, Peebles D, et al., 2004, Targeting the fetal respiratory muscles for prenatal gene therapy for Duchenne muscular dystrophy, 25th Annual Meeting of the Society-for-Maternal-Fetal-Medicine, Publisher: MOSBY-ELSEVIER, Pages: S22-S22, ISSN: 0002-9378
David A, Weisz B, Peebles D, et al., 2004, Ultrasound guided placement of an occlusive balloon in the trachea of fetal sheep, 25th Annual Meeting of the Society-for-Maternal-Fetal-Medicine, Publisher: MOSBY-ELSEVIER, Pages: S166-S166, ISSN: 0002-9378
Waddington SN, Nivsarkar MS, Mistry AR, et al., 2004, Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy, BLOOD, Vol: 104, Pages: 2714-2721, ISSN: 0006-4971
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- Citations: 100
Waddington SN, Kennea NL, Buckley SMK, et al., 2004, Fetal and neonatal gene therapy: benefits and pitfalls, 2nd European Conference and Practical Course on Towards Clinical Gene Therapy - Preclinical Gene Transfer Assessment, Publisher: NATURE PUBLISHING GROUP, Pages: S92-S97, ISSN: 0969-7128
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- Citations: 34
Coutelle C, 2004, In utero gene therapy for genetic disease: animal models, target diseases and ethical considertions, 2nd European Conference and Practical Course on Towards Clinical Gene Therapy - Preclinical Gene Transfer Assessment, Publisher: NATURE PUBLISHING GROUP, Pages: S143-S143, ISSN: 0969-7128
Waddington SN, Kennea NL, Buckley SMK, et al., 2004, Fetal and neonatal gene therapy: benefits and pitfalls., Gene Ther, Vol: 11 Suppl 1, Pages: S92-S97, ISSN: 0969-7128
The current approaches to gene therapy of monogenetic diseases into mature organisms are confronted with several problems including the following: (1) the underlying genetic defect may have already caused irreversible pathological changes; (2) the level of sufficient protein expression to ameliorate or prevent the disease requires prohibitively large amounts of gene delivery vector; (3) adult tissues may be poorly infected by conventional vector systems dependent upon cellular proliferation for optimal infection, for example, oncoretrovirus vectors; (4) immune responses, either pre-existing or developing following vector delivery, may rapidly eliminate transgenic protein expression and prevent future effective intervention. Early gene transfer, in the neonatal or even fetal period, may overcome some or all of these obstacles. The mammalian fetus enjoys a uniquely protected environment in the womb, bathed in a biochemically and physically supportive fluid devoid of myriad extra-uterine pathogens. Strong physical and chemical barriers to infection might, perhaps, impede the frenetic cell division. The physical support and the biochemical support provided by the fetal-maternal placental interface may, therefore, minimize the onset of genetic diseases manifest early in life. The fetal organism must prepare itself for birth, but lacking a mature adaptive immune system may depend upon more primordial immune defences. It is the nature of these defences, and the vulnerabilities they protect, that are poorly understood in the context of gene therapy and might provide useful information for approaches to gene therapy in the young, as well as perhaps the mature organism.
Waddington SN, Nivsarkar M, Mistry A, et al., 2004, Permanent phenotypic correction of Haemophilia B in immunocompetent mice by prenatal gene therapy, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: WILEY-BLACKWELL, Pages: S17-S17, ISSN: 1099-498X
Holder MV, Waddington SN, Gregory LG, et al., 2004, Comparison of gene expression from liver specific promoter/enhancer combinations in the context of a lentiviral backbone, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: JOHN WILEY & SONS LTD, Pages: S9-S9, ISSN: 1099-498X
David AL, Peebles D, Gregory L, et al., 2004, Towards prenatal gene therapy for cystic fibrosis: ultrasound guided delivery of recombinant adenoviral vectors to the fetal sheep trachea results in efficient marker gene expression in the airway epithelia, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: WILEY-BLACKWELL, Pages: S20-S21, ISSN: 1099-498X
Nivsarkar MS, Nathwani A, Mosley K, et al., 2004, Testosterone induced increase in transgene expression <i>in vivo</i> using Adenoviral Gene Delivery Vectors, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: WILEY-BLACKWELL, Pages: S12-S13, ISSN: 1099-498X
Vaysse L, Harbottle R, Bergau A, et al., 2004, Development of a self-assembling nuclear targeting vector system based on the tetracycline repressor protein, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: JOHN WILEY & SONS LTD, Pages: S15-S15, ISSN: 1099-498X
Harbottle R, Gregory L, Waddington S, et al., 2004, A mechanistic investigation of hydrodynamic mediated gene delivery, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: JOHN WILEY & SONS LTD, Pages: S9-S9, ISSN: 1099-498X
Gregory LG, Waddington SN, Holder M, et al., 2004, Pseudotyped lentiviral vectors:: Toward an applicability for <i>in utero</i> treatment of Duchenne Muscular Dystrophy, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: JOHN WILEY & SONS LTD, Pages: S18-S18, ISSN: 1099-498X
Bergau A, Gregory L, Waddington S, et al., 2004, Comparative analysis of the human cytokeratin K18 promoter with the CMV promoter by hydrodynamic injection of adult MF-1 mice, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: JOHN WILEY & SONS LTD, Pages: S5-S6, ISSN: 1099-498X
Bigger BW, Siapati KE, Mistry A, et al., 2004, Induction of immune tolerance and partial phenotypic correction of haermophiliac mice by lentiviral gene transfer of human factor IX to haematopoietic stem cells, 1st Annual Conference of the British-Society-for-Gene-Therapy, Publisher: JOHN WILEY & SONS LTD, Pages: S15-S15, ISSN: 1099-498X
Ward R, Coutelle C, 2004, Molecular and biochemical changes associated with acetaldehyde toxicity., 12th International Congress of the International-Society-for-Biomedical-Research-on-Alcoholism, Publisher: LIPPINCOTT WILLIAMS & WILKINS, Pages: 83A-83A, ISSN: 0145-6008
Coutelle C, Höhn B, Benesova M, et al., 2004, Increased breast cancer risk in alcohol consumers with the alcohol dehydrogenase 1C*1 allele., 12th International Congress of the International-Society-for-Biomedical-Research-on-Alcoholism, Publisher: LIPPINCOTT WILLIAMS & WILKINS, Pages: 14A-14A, ISSN: 0145-6008
Gregory LG, Waddington SN, Holder MV, et al., 2004, Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy, GENE THERAPY, Vol: 11, Pages: 1117-1125, ISSN: 0969-7128
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- Citations: 32
Bigger BW, Siapati EK, Mistry A, et al., 2004, Induction of immune tolerance and phenotypic rescue of hemophilic mice by stem cell mediated gene transfer, 7th Annual Meeting of the American-Society-of-Gene-Therapy, Publisher: ACADEMIC PRESS INC ELSEVIER SCIENCE, Pages: S64-S64, ISSN: 1525-0016
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