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Human Molecular Genetics, ISSN 0964-6906, 10/2019, Volume 28, Issue R1, pp. R102 - R107
Abstract Muscular dystrophy (MD) is a group of progressive genetic diseases affecting the musculature that are characterized by inflammatory infiltrates,... 
CT GALNAC TRANSFERASE | IN-VIVO CORRECTION | SKELETAL-MUSCLE | OVEREXPRESSION | UTROPHIN | PROTEIN | RNA | BIOCHEMISTRY & MOLECULAR BIOLOGY | MOUSE MODEL | GROWTH | GENETICS & HEREDITY | MYOTONIC-DYSTROPHY
Journal Article
Journal Article
Blood, ISSN 0006-4971, 12/2014, Volume 124, Issue 21, pp. 105 - 105
Abstract The paired basic amino acid cleaving enzyme (PACE)/Furin is a protein convertase system that plays a vital role in several biological processes,... 
Journal Article
Blood, ISSN 0006-4971, 11/2013, Volume 122, Issue 21, pp. 4203 - 4203
Abstract Emerging data from early phase clinical studies of AAV gene therapy for hemophilia B (HB) (factor IX [FIX] deficiency) show sustained expression of... 
Journal Article
01/2015, ISBN 1321850670
Treatment of hemophilia, which involves infusion of the missing clotting factor, is often hindered by the development of neutralizing antibodies to the... 
Molecular biology | Oncology | Immunology
Dissertation
Blood, ISSN 0006-4971, 11/2012, Volume 120, Issue 21, pp. 3350 - 3350
Abstract Abstract 3350 Cancer is frequently associated with activation of coagulation, and a procoagulant state facilitates tumor metastasis. Recent studies... 
Journal Article
Blood, ISSN 1528-0020, 12/2010, Volume 116, Issue 26, pp. 5842 - 5848
Inhibitory antibodies to factor VIII (FVIII) are a major complication in the treatment of hemophilia A, affecting approximately 20% to 30% of patients. Current... 
Journal Article
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