Safety and in vivo expression of a GNE-transgene: a novel treatment approach for hereditary inclusion body myopathy-2.

Hereditary inclusion body myopathy-2 (HIBM2) is an adult-onset, muscular disease caused by mutations in the GNE gene. HIBM2-associated GNE mutations causing hyposialyation have been proposed to contribute to reduced muscle function in patients with HIBM2, though the exact cause of this disease is unknown. In the current studies we examined pre-clinical in vivo toxicity, and expression of the plasmid-based, CMV driven wild-type GNE plasmid vector.

Evaluation of GFR estimating equations in the general community: implications for screening.

The Kidney Disease Outcomes Quality Initiative has recommended the use of GFR estimating equations to detect silent chronic kidney disease (CKD) in the community. The benefit of general reporting of CKD must be balanced with the harm of mislabeling people who do not have CKD. The popular Cockcroft-Gault (CG) and Modification of Diet in Renal Disease (MDRD) GFR estimating equations were compared with the recently devised Rule equation in a representative community population sample (2166) divided into subsamples with (385) and without (1781) previous renal impairment.

Optimal timing of initiation of chronic hemodialysis?

Despite the availability of clinical guidelines for the timing of dialysis initiation in both the United States and Canada, patients continue to start dialysis at very low levels of predicted glomerular filtration rate (GFR). A cross-sectional study was performed to determine the demographic and clinical characteristics of patients who started hemodialysis, their level of GFR, and mortality at 1 and 2 years following the initiation of dialysis. Retrospective data were collected on all eligible patients who commenced chronic hemodialysis in 1 tertiary care center in Canada from March 2001 to February 2005.