Improved efficacy of FKRP AAV gene therapy by combination with ribitol treatment for LGMD2I.

Mutations in the fukutin-related protein (FKRP) gene cause dystroglycanopathy, with disease severity ranging from mild LGMD2I to severe congenital muscular dystrophy. Recently, considerable progress has been made in developing experimental therapies, with adeno-associated virus (AAV) gene therapy and ribitol treatment demonstrating significant therapeutic effect. However, each treatment has its strengths and weaknesses.

Ribitol dose-dependently enhances matriglycan expression and improves muscle function with prolonged life span in limb girdle muscular dystrophy 2I mouse model.

Limb Girdle Muscular Dystrophy 2I (LGMDR9) is one of the most common LGMD characterized by defects in glycosylation of α-dystroglycan (matriglycan) resulting from mutations of Fukutin-related protein (FKRP). There is no effective therapy currently available. We recently demonstrated that ribitol supplement increases levels of matriglycan in cells in vitro and in FKRP-P448L (P448L) mutant mouse model through drinking water administration.

In vitro cytotoxicity of a low-shrinkage polymerizable liquid crystal resin monomer.

The objective of this study was to determine the in vitro cytotoxicity of novel, polymerizable liquid crystal resin monomers when placed in direct contact with dental and nondental cell lines. One common dimethacrylate and three liquid crystal compounds, Bis-glycidyl methacrylate (Bis-GMA), 2-(t-butyl)-1,4-bis-{4-(6-acryloxy-hexane-1-oxy)-benzoyloxy}-benzene (C6), 2-(t-butyl), 1-[6-(3-acryloxy-propionoxy)-hexane-1-oxy-benzoyloxy], 4-[4-(6-acryloxy-hexane-1-oxy)-benzoyloxy]-benxene (by-product), and a 3:2 mixture of C6 and by-product, respectively, were tested for relative cytotoxicity in vitro. Cultured dental and nondental cells were treated for 24 h with test compound dissolved in media over a fourfold range of concentration (10(-4) -10(-7) mol/L).

Viral hepatitis in minority America.

Viral hepatitis continues as an important public health concern in the United States. Available data indicate that acute and chronic viral hepatitis remains an important cause of morbidity and mortality in this country despite the availability of immunization for hepatitis A and B and pharmacologic therapy for chronic hepatitis B and C. Minority populations within the United States are disproportionately affected by acute and chronic viral hepatitis.