Zobrazeno 1 - 10 of 10 pro vyhledávání: '"Jacqueline Brassard"'
1
Akademický článek
Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity
Autor: Michelle Eggers, Charles H Vannoy, Jianyong Huang, Pravinkumar Purushothaman, Jacqueline Brassard, Carlos Fonck, Hui Meng, Mariah J Prom, Michael W Lawlor, Justine Cunningham, Chanchal Sadhu, Fulvio Mavilio
Publikováno v: EMBO Molecular Medicine, Vol 14, Iss 1, Pp 1-15 (2021)
Abstract Pompe disease is a severe disorder caused by loss of acid α‐glucosidase (GAA), leading to glycogen accumulation in tissues and neuromuscular and cardiac dysfunction. Enzyme replacement therapy is the only available treatment. AT845 is an
Externí odkaz: https://doaj.org/article/35fec66cd46f45f6b1e570648c4fe7cb
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2
Muscle‐directed gene therapy corrects Pompe disease and uncovers species‐specific GAA immunogenicity
Autor: Charles H Vannoy, Carlos Fonck, Mariah J. Prom, Pravinkumar Purushothaman, Chanchal Sadhu, Michelle Eggers, Fulvio Mavilio, Jacqueline Brassard, Justine Cunningham, Hui Meng, Michael W. Lawlor, Jianyong Huang
Publikováno v: EMBO Molecular Medicine, Vol 14, Iss 1, Pp n/a-n/a (2022)
EMBO Molecular Medicine
Pompe disease is a severe disorder caused by loss of acid α‐glucosidase (GAA), leading to glycogen accumulation in tissues and neuromuscular and cardiac dysfunction. Enzyme replacement therapy is the only available treatment. AT845 is an adeno‐a
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8577a552e15ca514b290d0b06e77bb0e
https://doaj.org/article/35fec66cd46f45f6b1e570648c4fe7cb
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3
Immunology and Pathology in Ocular Drug Development
Autor: Meg Ferrell Ramos, Sharmila Masli, Jacqueline Brassard
Publikováno v: Toxicologic pathology. 49(3)
Clear vision is dependent on features that protect the anatomical integrity of the eye (cornea and sclera) and those that contribute to internal ocular homeostasis by conferring hemangiogenic (avascular tissues and antiangiogenic factors), lymphangio
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::61d87b1b916615e15953aa720ef734b4
https://pubmed.ncbi.nlm.nih.gov/33468035
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6
Safety Evaluation of Ocular Drugs
Autor: Jacqueline Brassard, Chang Vangyi, Steven S. Matsumoto, Mathilde Stern, M. Ferrell Ramos, Andrea S. Kim, Mayssa Attar
Toxicology studies should be properly designed so that valid conclusions can be made without wasting precious resources. Statistical tests to analyze data from these studies should be implemented to properly account for the experimental design. To ap
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_________::99668cb48ae90e5bba33fb4ff2d22720
https://doi.org/10.1016/b978-0-12-803620-4.00029-3
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8
Review of continuing education course on hemostasis
Autor: Dale C. Baker, Jacqueline Brassard
Publikováno v: Toxicologic pathology. 39(1)
The continuing education course “Hemostasis” provided a comprehensive review of hemostasis and selected perturbations of the underlying processes as well as an assessment of hemostasis in animal models and preclinical testing environments. The se
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e30e7fee08432e87d0103fb743bdd962
https://pubmed.ncbi.nlm.nih.gov/21131603
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9
Expression and characterization of von Willebrand factor dimerization defects in different types of von Willebrand disease
Autor: Johannes Oldenburg, Zaverio M. Ruggeri, Ulrich Budde, Rainer Schwaab, Kerstin Mainusch, Tobias Obser, Jacqueline Brassard, Sonja Schneppenheim, Reinhard Schneppenheim
Publikováno v: Blood. 97(7)
Dimerization defects of von Willebrand factor (vWF) protomers underlie von Willebrand disease (vWD) type 2A, subtype IID (vWD 2A/IID), and corresponding mutations have been identified at the 3′ end of the vWF gene in exon 52. This study identified
Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::778a97e28457bd3187b2dc4a49885955
https://pubmed.ncbi.nlm.nih.gov/11264172
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10
Akademický článek
Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity.
Autor: Eggers M; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Vannoy CH; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Huang J; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Purushothaman P; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Brassard J; Jacqueline Brassard Toxicologic Pathology Consulting, Tustin, CA, USA., Fonck C; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Meng H; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA., Prom MJ; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA., Lawlor MW; Department of Pathology and Neuroscience Research Center, Medical College of Wisconsin, Milwaukee, WI, USA., Cunningham J; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Sadhu C; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA., Mavilio F; Nonclinical, Pharmacology/Toxicology, Audentes Therapeutics, San Francisco, CA, USA.; Department of Life Sciences, University of Modena and Reggio Emilia, Modena, Italy.
Publikováno v: EMBO molecular medicine [EMBO Mol Med] 2022 Jan 11; Vol. 14 (1), pp. e13968. Date of Electronic Publication: 2021 Dec 01.
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