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CLINICAL TRIAL OF GENE THERAPY FOR MPS VI. A SEVERE LYSOSOMAL STORAGE DISORDER. Enzyme replacement therapy, the current treatment for MPS VI, requires weekly infusions of a costly enzyme and has limited efficacy on bone and corneal disease. Based on encouraging preclinical results, gene therapy based on a single intravascular administration of adeno-associated viral (AAV) vectors targeting liver has the potential to provide a lifelong source of ARSB. The results from this clinical trial proposed by the M...

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Welcome to MeuSIX — .: The MeuSIX Project :. | meusix.tigem.it Reviews

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CLINICAL TRIAL OF GENE THERAPY FOR MPS VI. A SEVERE LYSOSOMAL STORAGE DISORDER. Enzyme replacement therapy, the current treatment for MPS VI, requires weekly infusions of a costly enzyme and has limited efficacy on bone and corneal disease. Based on encouraging preclinical results, gene therapy based on a single intravascular administration of adeno-associated viral (AAV) vectors targeting liver has the potential to provide a lifelong source of ARSB. The results from this clinical trial proposed by the M...

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Welcome to MeuSIX — .: The MeuSIX Project :. | meusix.tigem.it Reviews

https://meusix.tigem.it

CLINICAL TRIAL OF GENE THERAPY FOR MPS VI. A SEVERE LYSOSOMAL STORAGE DISORDER. Enzyme replacement therapy, the current treatment for MPS VI, requires weekly infusions of a costly enzyme and has limited efficacy on bone and corneal disease. Based on encouraging preclinical results, gene therapy based on a single intravascular administration of adeno-associated viral (AAV) vectors targeting liver has the potential to provide a lifelong source of ARSB. The results from this clinical trial proposed by the M...

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meusix.tigem.it

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.: The MeuSIX Project :.

http://meusix.tigem.it/mail_password_form

For security reasons, we store your password encrypted, and cannot mail it to you. If you would like to reset your password, fill out the form below and we will send you an email at the address you gave when you registered to start the process of resetting your password. My user name is. If this will not work for you (for example, if you forgot your user name or your email address has changed), contact the site administration. This is Schools Diazo Plone Theme.

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Work packages — .: The MeuSIX Project :.

http://meusix.tigem.it/meusix-project/work-packages

Production of clinical-grade AAV2/8.TGB.hARSB. Pre-clinical evaluation of the AAV2/8.TBG.hARSB GMP vector. Multicentre Phase I/II MPS VI clinical trial. Ethics and Patient Organizations. Cookies are not enabled. You must enable cookies before you can log in. This is Schools Diazo Plone Theme.

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Flyer — .: The MeuSIX Project :.

http://meusix.tigem.it/meusix-project/documentation

Clinical trial MeuSIX-July2014.pdf. PDF document, 5808 kB (5948288 bytes). Cookies are not enabled. You must enable cookies before you can log in. This is Schools Diazo Plone Theme.

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Partners — .: The MeuSIX Project :.

http://meusix.tigem.it/meusix-project/participants

UNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO II. ERASMUS UNIVERSITAIR MEDISCH CENTRUM ROTTERDAM. UNIVERSITA' DEGLI STUDI DI MILANO-BICOCCA. Cookies are not enabled. You must enable cookies before you can log in. This is Schools Diazo Plone Theme.

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Meetings and Events — .: The MeuSIX Project :.

http://meusix.tigem.it/meusix-project/meetings-and-events

January 24, 2013. March 20, 2013. MPS VI patients presentation meeting. November 4-5, 2013. Ethics Advisory Group meeting. March 10, 2014. March 27, 2015. March 14, 2016. Cookies are not enabled. You must enable cookies before you can log in. This is Schools Diazo Plone Theme.

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Collaborations and Partnerships | Genosafe

http://www.genosafe.com/about-us/collaborations-and-partnerships

A full CRO and consulting company. Careers – Jobs. Professional and scientific associations. QC testing – Product characterization. Call or visit us. Careers – Jobs. Professional and scientific associations. Participating in in collaborative projects are excellent opportunities for GenoSafe to strengthen its research and innovation activities. Transposon-based, targeted gene therapy to treat age-related macular degeneration. Coordinator: Hôpitaux Universitaires de Genève. This project is focused on the c...

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Projects - INFORMAPRO

http://www.informapro.it/projects.html

CME and academic education. CLINICAL TRIAL OF GENE THERAPY FOR MPS VI. 8203;A SEVERE LYSOSOMAL STORAGE DISORDER. I-PRO is partner of the MeuSIX FP7 research project, under the coordination of Fondazione Theleton. The MeuSIX consortium plans to conduct a multicenter phase 1/2 clinical trial to investigate the safety and efficacy of AAV-mediated gene therapy in patients with MPS VI. Moreover, the approach developed may facilitate the development of similar approaches for other inborn errors of metabolism.

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meusix.tigem.it

Welcome to MeuSIX — .: The MeuSIX Project :.

CLINICAL TRIAL OF GENE THERAPY FOR MPS VI. A SEVERE LYSOSOMAL STORAGE DISORDER. Enzyme replacement therapy, the current treatment for MPS VI, requires weekly infusions of a costly enzyme and has limited efficacy on bone and corneal disease. Based on encouraging preclinical results, gene therapy based on a single intravascular administration of adeno-associated viral (AAV) vectors targeting liver has the potential to provide a lifelong source of ARSB. The results from this clinical trial proposed by the M...

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