Development of a Novel AAV Gene Therapy Cassette with Improved Safety Features and Efficacy in a Mouse Model of Rett Syndrome.

Rett syndrome (RTT), caused by loss-of-function mutations in the gene, is a neurological disorder characterized by severe impairment of motor and cognitive functions. The aim of this study was to investigate the impact of vector design, dosage, and delivery route on the efficacy and safety of gene augmentation therapy in mouse models of RTT. Our results show that AAV-mediated delivery of to null mice by systemic administration, and utilizing a minimal endogenous promoter, was associated with a narrow therapeutic window and resulted in liver toxicity at higher doses.

Clinical efficacy of pioglitazone: generic vs. original product.

Background: Pioglitazone, an oral antidiabetic agent in the class of thiazolidinediones (TZDs), was widely used in the case of insulin tolerance as it provided more benefit to patients with type 2 diabetes. However, the original product is costly while some generic products are available at the substantial lower cost in Thailand. The objective of the present study was to assess bioequivalence in terms of efficacy between generic and original pioglitazone products.

Herbal medicine: affecting factors and prevalence of use among Thai population in Bangkok.

Background: Remarkable growth in use of herbal medicines has recently been noted. In Thailand, eight items of herbal medicines with single composition are currently placed on the National List of Essential Medicines (EM). This study was to clarify the actual state of factors affecting the usage and knowledge of these herbal medicines, as the study concerning with these aspects was infrequently performed.

Adverse effect of pioglitazone in military personnel and their families: a preliminary report.

Background: Thiazolidinediones (rosiglitazone and pioglitazone) whether administered alone or in combination with metformin, sulfonylurea, or insulin, are often accompanied by an increase in weight and/or plasma volume. Several studies have shown the adverse effect of weight gain and edema with rosiglitazone. But there was less data with pioglitazone, especially in military personnel and their families.