Permanent phenotypic correction of Haemophilia B in immunocompetent mice by prenatal gene therapy Running title: Permanent correction of haemophilia in a fetal mouse model

نویسندگان

  • Simon N. Waddington
  • Megha S. Nivsarkar
  • Ajay R. Mistry
  • L. Mosley
  • Kyriacos Mitrophanous
  • Pippa Radcliffe
  • Maxine V. Holder
  • Mairi Brittan
  • Anastasios Georgiadis
  • Faisal Al-Allaf
  • Brian W. Bigger
  • Terence Cook
  • R. Ali
  • Adrian Thrasher
چکیده

Gene Therapy Research Group, Section of Cell and Molecular Biology, Imperial College London, London, SW7 2AZ, UK Molecular Immunology Unit, Institute of Child Health, University College London, London, WC1N 1EH, UK Haemostasis and Thrombosis, MRC Clinical Sciences Centre, Imperial College London, London, W12 0NN, UK Renal Medicine Section, Faculty of Medicine, Imperial College London, London, W12 0NN Oxford BioMedica (UK) Ltd, Oxford, OX4 4GA, UK Histopathology Unit, Cancer Research UK, London UK, WC2A 3PX Department of Molecular Genetics, Institute of Ophthalmology, University College London, London, EC1V 9EL, UK

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Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy.

Hemophilia B, also known as Christmas disease, arises from mutations in the factor IX (F9) gene. Its treatment in humans, by recombinant protein substitution, is expensive, thus limiting its application to intermittent treatment in bleeding episodes and prophylaxis during surgery; development of inhibitory antibodies is an associated hazard. This study demonstrates permanent therapeutic correct...

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GENE THERAPY Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy

Hemophilia B, also known as Christmas disease, arises from mutations in the factor IX (F9) gene. Its treatment in humans, by recombinant protein substitution, is expensive, thus limiting its application to intermittent treatment in bleeding episodes and prophylaxis during surgery; development of inhibitory antibodies is an associated hazard. This study demonstrates permanent therapeutic correct...

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O-45: Quantification of Cell-Free-Fetal-DNAfrom Maternal Plasma for the First Time in Pakistan:Implications for Non-Invasive PrenatalDiagnosis of Genetic Disorders

Background: Current prenatal diagnosis requires invasive testing which carries a 1-4% procedure-related-risk of miscarriage; hence, non-invasive techniques are desired. The recent demonstration of cell-free-fetal-DNA enriched from maternal plasma has opened new possibilities for non-invasive-prenatal-diagnosis of not only genetic-disorders such as β-thalassaemia and haemophilia but also chromos...

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O-45: Quantification of Cell-Free-Fetal-DNAfrom Maternal Plasma for the First Time in Pakistan:Diagnosis of Genetic Disorders

Background: Current prenatal diagnosis requires invasive testing which carries a 1-4% procedure-related-risk of miscarriage; hence, non-invasive techniques are desired. The recent demonstration of cell-free-fetal-DNA enriched from maternal plasma has opened new possibilities for non-invasive-prenatal-diagnosis of not only genetic-disorders such as β-thalassaemia and haemophilia but also chromos...

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تاریخ انتشار 2004