Chapter 20 - Advanced drug delivery systems involving lysosomal storage disorders for Schinder disease and other disorders

Abstract

Lysosomal storage diseases (LSDs) include a broad spectrum of 50 discrete genetic diseases, primarily due to defective lysosomal protein or their activity; in other scenarios, the irregularities arise from non-lysosomal activities such as lysosomal biogenesis or protein maturation. Gaucher disease is the most common disease, followed by Fabry disease. The prevalence of LSDs worldwide has reached epidemic proportions. Most LSDs are inherited as autosomal recessive traits due to mutations in genes encoding lysosomal proteins, which elicits a pathogenetic cascade. However, statistics indicate the incidence of each LSDs is very rare per se, ranging from 1 in 50,000 to 1 in 250,000 live births, but LSDs collectively are more common disorders (1:5000 live births). By far, various methodologies have been adopted besides systemic drug therapy. However, in many cases, surgeries are overused for preventing or alleviating the conditions. Conversely, the currently available therapies remain inadequate or lack effectiveness, especially in terms of patient compliance. Henceforth it remains crucial to explore various advanced therapies and delivery systems to treat better and lessen side effects. This chapter emphasizes potential targets available for LSDs and recent advances in delivery systems and subsequent novel therapies. Furthermore, we highlighted the significance of gene therapy, focusing on viral vectors; also, we stressed the future roadmap for gene therapy for LSDs. Additionally, current studies in clinical trials that potentially explored alleviating LSDs are briefly mentioned in this chapter.