Sphingolipids as Emerging Drug Targets: Therapeutic Applications of Ceramide Analogs

Human diseases caused by alterations in the metabolism of sphingolipids or glycosphingolipids are mainly disorders of the degradation of these compounds. The sphingolipidoses are a group of monogenic inherited diseases caused by defects in the system of lysosomal sphingolipid degradation, with subsequent accumulation of non-degradable storage material in one or more organs. Most sphingolipidoses are associated with high mortality. Both, the ratio of substrate influx into the lysosomes and the reduced degradative capacity can be addressed by therapeutic approaches. In addition to symptomatic treatments, the current strategies for restoration of the reduced substrate degradation within the lysosome are enzyme replacement therapy (ERT), cell-mediated therapy (CMT) including bone marrow transplantation (BMT) and cell-mediated "cross correction", gene therapy, and enzyme-enhancement therapy with chemical chaperones. The reduction of substrate influx into the lysosomes can be achieved by substrate reduction therapy. Patients suffering from the attenuated form (type 1) of Gaucher disease and from Fabry disease have been successfully treated with ERT.

show abstract

Sphingolipid metabolism diseases

Kolter

Sandhoff

2006

Biochimica et Biophysica Acta (BBA) - Biomembranes

323

261

View full text Add to dashboard Cite

show abstract

Synthesis of novel ceramide-like penetration enhancers

Takacs

Bubenyák

Váradi

et al. 2011

Tetrahedron Letters

View full text Add to dashboard Cite

scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.

Contact Info

customersupport@researchsolutions.com

10624 S. Eastern Ave., Ste. A-614

Henderson, NV 89052, USA

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

Blog Terms and Conditions API Terms Privacy Policy Contact Cookie Preferences Do Not Sell or Share My Personal Information

Made with 💙 for researchers

Part of the Research Solutions Family.

Sphingolipids as Emerging Drug Targets: Therapeutic Applications of Ceramide Analogs

Cited by 2 publications

References 0 publications

Sphingolipid metabolism diseases

Sphingolipid metabolism diseases

Synthesis of novel ceramide-like penetration enhancers

Contact Info

Product

Resources

About