Source enbrel european public assessment report

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Source: Enbrel®, European Public Assessment Report Indications of etanercept rheumatoid arthritis juvenile idiopathic arthritis ankylosing spondylitis psoriatic arthritis plaque psoriasis paediatric plaque psoriasis
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Vaccines 23 Prophylactic vaccines against infectious diseases e.g. Hepatitis B vaccination Therapeutic vaccines e.g. cancer vaccines intensive research is ongoing Production derived from natural source recombinant DNA technology
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Blood and blood components 24 For the treatment of anaemia thrombocytopenia clotting deficiencies Blood components red blood cells white blood cells plasma clotting factors platelets Whole blood
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Allergenic products 25 Application used for specific immunotherapy (SIT) in vivo diagnosis of immunoglobulin E (IgE)-mediated allergic diseases E.g. Prick skin allergy test
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Gene therapy 26 First gene therapy approved in EU Recurrent pancreatitis (lipoprotein lipase deficiency) Vector: adeno-associated virus Source: European Medicines Agency homepage „contain genes that lead to a therapeutic effect. They work by inserting 'recombinant' genes into cells , usually to treat a variety of diseases, including genetic disorders , cancer or long-term diseases . A recombinant gene is a stretch of DNA that is created in the laboratory, bringing together DNA from different sources”
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The history of the gene therapy The history of the gene therapy 1977 - A gene was successfully delivered into mammalian cells 1990 - First human gene therapy was approved: SCID 1999 - J. Gelsinger with OTC deficiency died from organ failure after gene therapy 2000 - A. Fischer cured children with SCID using retroviral vector, 2 of the children developed leukemia. FDA halted the use of retroviruses in the US 2006 - Patients was successfully treated with metastatic melanoma using killer T cells genetically retargeted to attack the cancer cells 2006 - Succesfull gene-based th. for the treatment of HIV: lentiviral vector for delivery of an antisense gene againts HIV envelope 2009 - Researchers succeeded at halting adrenoleukodystrophy, using a vector derived from HIV to deliver the gene for the missing enzyme
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The number of gene therapeutical clinical trials The number of gene therapeutical clinical trials 2012 N:3398 2006 N:1064
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General applications of General applications of therapeutic therapeutic gene transfer gene transfer 1. Molecular therapy for genetic diseases 2. Establishment of a stable gene reservoir as a source of therapeutic proteins in non - genetic diseases 3 . . Destruction of malignant cells in neoplasias 4. DNA vaccines
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20-02-14 Vectors Viral Nonviral Plasmid Arteficial Chr Liposomes DNAsomes Nanoparticlees Retro Herpes Adeno AAV Lenti Strategies Gene delivery
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Molecular therapies Molecular therapies DNA modulating therapies Gene replacement – to replace the defected gene Gene transfer – to upregulate therapeutic protein Gene editing – to correct the defected gene Gene shifting – to upregulate healthy mtDNA molecules, change ratio of heteroplasmy RNA modulating therapies
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