M
Mark E. Haskins
Researcher at University of Pennsylvania
Publications - 205
Citations - 7516
Mark E. Haskins is an academic researcher from University of Pennsylvania. The author has contributed to research in topics: Mucopolysaccharidosis & Lysosomal storage disease. The author has an hindex of 46, co-authored 202 publications receiving 7115 citations. Previous affiliations of Mark E. Haskins include North Shore University Hospital & National Institutes of Health.
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Journal ArticleDOI
Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease
TL;DR: The data suggest that gene transfer mediated by adeno-associated virus can achieve therapeutically relevant levels of enzyme very early in life and that the rapid growth and differentiation of tissues does not limit long-term expression.
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Mechanism of Glycosaminoglycan-Mediated Bone and Joint Disease Implications for the Mucopolysaccharidoses and Other Connective Tissue Diseases
Calogera M. Simonaro,Marina D'Angelo,Xingxuan He,Efrat Eliyahu,Nataly Shtraizent,Mark E. Haskins,Edward H. Schuchman +6 more
TL;DR: Analysis of MPS rats, cats, and/or dogs revealed that MPS synovial fibroblasts and fluid displayed elevated expression of numerous inflammatory molecules, including several proteins important for lipopolysaccharide signaling, and further defined the mechanism of GAG-stimulated disease.
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Joint and Bone Disease in Mucopolysaccharidoses VI and VII: Identification of New Therapeutic Targets and BioMarkers Using Animal Models
TL;DR: It is found that the stimulation of MPS connective tissue cells by the inflammatory cytokines causes enhanced secretion of several matrix-degrading metalloproteinases (MMPs), and this information should aid in the evaluation of existing therapies for these disorders and may lead to the development of new therapeutic approaches.
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Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs
Katherine P. Ponder,John R. Melniczek,Lingfei Xu,Margaret A. Weil,Thomas O'Malley,Patricia O'Donnell,Van W. Knox,Gustavo D. Aguirre,Hamutal Mazrier,N. Matthew Ellinwood,Margaret M. Sleeper,Albert M. Maguire,Susan W. Volk,Robert L Mango,Jean Zweigle,John H. Wolfe,Mark E. Haskins +16 more
TL;DR: This is the first successful application of gene therapy in preventing the clinical manifestations of a lysosomal storage disease in a large animal.
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Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model.
Valder R. Arruda,Hansell H. Stedman,Timothy C. Nichols,Mark E. Haskins,Matthew Nicholson,Roland W. Herzog,Linda B. Couto,Katherine A. High +7 more
TL;DR: This translational study provides the first report of long-term, robust Factor IX expression (circulating levels of 4%-14%) by muscle-directed gene transfer in a large animal, resulting in essentially complete correction of the bleeding disorder in hemophilic dogs.