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Applied genetics / Viruses / Biotechnology / Viral vector / Gene therapy / Adeno-associated virus / Vector / Retrovirus / Oncolytic virus / Biology / Gene delivery / Molecular biology


F E AT U R E Gene therapy finds its niche Cormac Sheridan Gene therapy is finally poised to make a contribution to the treatment of debilitating, highly penetrant genetic diseases that have proved intractable to other r
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Document Date: 2012-03-01 05:18:48

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Europe / /

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University of Florida’s Snyder / SaintVincent de Paul Hospital / clinic The / University Paris Descartes SCID-X1 / University of Florida / Necker Hospital / Children’s National Medical Center / University of California / Massachusetts Institute of Technology / Salk Institute / University of North Carolina / In complex / San Raffaele Telethon Institute / University Paris Descartes / University of Pennsylvania / /

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biotechnology volume / important manufacturing / therapy for Parkinson’s disease / Adherent cell systems / biotechnology / therapy for rare diseases / treatment of debilitating / highly penetrant genetic diseases / gene delivery protocols / treatment for head and neck cancer / vector technology / large pharmaceutical / downstream processing / biotechnology feature / standardized protocols / immune systems / insertion site / manufacturing practices / gas station / plasmid technology / assistive technologies / /

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HIV / p53-deficient tumor / murine leukemia / malignant and benign tumors / hyperargininemia / human RPE65 Congenital amaurosis / leukemia / tumor / lung inflammation / genetic disorder / osteosarcoma / chronic angina / malignant glioma / murine leukemia virus / Parkinson’s disease / diseases / pancreatitis / monogenic disorders / HIV/AIDS / Vaccinia virus / deficiency / certain retinal disorders / vesicular stomatitis virus / slight paralysis / chimeric T-cell receptor gene B-cell leukemia / X-linked immune disorder / severe combined immune deficiency / Herpes / Aldrich syndrome / hemiparesis / demyelination / head and neck cancer / blindness / ultra-rare disorder / disease / pain / ADA deficiency / highly penetrant genetic diseases / metachromatic leukodystrophy / multifactorial diseases / rare diseases / seizures / successful pulmonary delivery / Leber’s congenital amaurosis / Melanoma / severe inflammatory response / lipoprotein lipase deficiency / replication-defective herpes simplex virus / nitrogen metabolism disorder ornithine transcarbamylase / mesothelioma Colon cancer / cancer / complex biology underlying disease / thyroid tumors / severe genetic disease / subsequently developed leukemia / X-linked adrenoleukodystrophy / X-linked SCID / recurrent infections / Congenital amaurosis / cystic fibrosis transmembrane conductance regulator / cystic fibrosis / nitrogen metabolism disorder hyperargininemia / specific diseases / severe combined immunodeficiency / degenerative disorder / rare disease / p53 tumor / SCID / Adenovirus AAV Retrovirus/lentivirus Family Adenoviridae Parvoviridae Retroviridae Herpesvirus Herpesviridae Genome dsDNA ssDNA ssRNA+ dsDNA Infection / inflammation / /

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surgery / blood transfusions / HAART / antiviral therapy / Gene therapy / enzyme replacement therapy / amputation / radiation / bone marrow transplant / /

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L3 / /

Organization

Massachusetts Institute of Technology / Subsequent administration / the University of Pennsylvania / Philadelphia / Children’s National Medical Center / French Muscular Dystrophy Association / Salk Institute / Committee for Human Medicinal Products / Minneapolis Heart Institute Foundation / San Raffaele Telethon Institute for Gene Therapy / Necker Hospital for Sick Children / Oxford University / National Institute of Health / University of North Carolina / European Medicines Agency / US Food and Drug Administration / University of California / Los Angeles / University of Florida / /

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Daniel Anderson / Luigi Naldini / Claudio Bordignon / Stuart Naylor / Gaithersburg / Patrick Aubourg / Jean Bennett / Frederic Revah / Marina Cavazzana-Calvo / Alain Fisher / Darren Wolfe / Alessandro Aiuti / Philippe Leboulch / Martin Cline / Jeff Ostrove / Carl June / Timothy Henry / Michael Blaese / Gene Ther / ADA-SCID GT / Leaf Huang / Fulvio Mavillo / Hyde / Sunway Biotech / Robert Langer / Boro Dropulic / Deborah Gill / Richard Snyder / Alain Fischer / Nick Leschly / Jörn Aldag / James Wilson / Ashanti DeSilva / Jesse Gelsinger / /

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first external player / freelance writer / CEO / LPL deficiency Filed Aventis Paris Ad5CMV-p53 Head / director and lead investigator / treating head / president and CEO / director / co-leader / chair / CEO of US subsidiary / /

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