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pro vyhledávání: '"Thomas Lester"'
Autor:
Xin Chen, Daniel A. Lim, Michael W. Lawlor, David Dimmock, Charles H. Vite, Thomas Lester, Fatemeh Tavakkoli, Chanchal Sadhu, Suyash Prasad, Steven J. Gray
Publikováno v:
Human Gene Therapy. 34:94-111
Autor:
Xin, Chen, Daniel A, Lim, Michael W, Lawlor, David, Dimmock, Charles, Vite, Thomas, Lester, Fatemeh, Tavakkoli, Chanchal, Sadhu, Suyash, Prasad, Steven J, Gray
Publikováno v:
Human gene therapy.
Adeno-associated virus (AAV)-based gene therapies, exemplified by the approved therapy for spinal muscular atrophy, have the potential to deliver disease-course-altering treatments for central nervous system (CNS) indications. However, several clinic
Publikováno v:
2018 ASEE Annual Conference & Exposition Proceedings.
Publikováno v:
Pharmaceutical Regulatory Affairs: Open Access.
Delivery of therapeutic agents directly to the central nervous system can be critical to address a number of diseases. Intraventricular administration of drugs has been used for over 50 years. Despite a substantial number of drugs routinely administe
Autor:
Irene Slavc, Thomas Lester, Daniel A. Lim, Manfred Westphal, Jessica L. Cohen-Pfeffer, Adam J. Shaywitz, Sridharan Gururangan
Publikováno v:
Pediatric Neurology.
Intrathecal delivery methods have been used for many decades to treat a broad range of central nervous system disorders. A literature review demonstrated that intracerebroventricular route is an established and well-tolerated method for prolonged cen
Autor:
Merry Passage, Emil D. Kakkis, Steven Q. Le, Maryn Peinovich, A. W. Krieger, Thomas Lester, Patricia I. Dickson
Publikováno v:
Journal of Inherited Metabolic Disease. 32:253-258
Intravenous enzyme replacement therapy with recombinant human α-L-iduronidase (rhIDU) is used weekly to treat mucopolysaccharidosis (MPS) I. We tested continuous administration of rhIDU at two dosing levels (0.58 mg/kg per week and 2 mg/kg per week)
Autor:
Bin Zhao, Jeffrey F. Lemontt, Pascale M.N. Tiger, Brigette L. Tippin, Steven Q. Le, Javier Femenia, Emil D. Kakkis, Thomas Lester, Patricia I. Dickson, Merry Passage, Scott Clarke, Moin Vera
Publikováno v:
JIMD Reports ISBN: 9783642334320
The immune response to exogenous protein has been shown to reduce therapeutic efficacy in animal models of enzyme replacement therapy. A previously published study demonstrated an immunosuppressive regimen which successfully induced immune tolerance
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cf75b41f65bc0b8fc22cc621602cc7b7
https://doi.org/10.1007/8904_2012_162
https://doi.org/10.1007/8904_2012_162
Publikováno v:
Journal of veterinary diagnostic investigation : official publication of the American Association of Veterinary Laboratory Diagnosticians, Inc. 22(4)
Epizootic hemorrhagic disease virus (EHDV) is a significant pathogen of wild and sometimes domestic ungulates worldwide. Rapid and reliable methods for virus detection and identification play an essential part in the control of epizootic hemorrhagic
Autor:
Patricia I. Dickson, Hayden Manuel, Catherine Jabagat, Emil D. Kakkis, Maryn Peinovich, Thomas Lester, Steven Q. Le, Merry Passage, Aimee Krieger, Michael F. McEntee
Publikováno v:
Journal of Clinical Investigation.
Mucopolysaccharidoses (MPSs) are lysosomal storage diseases caused by a deficit in the enzymes needed for glycosaminoglycan (GAG) degradation. Enzyme replacement therapy with recombinant human α-l-iduronidase successfully reduces lysosomal storage i