Lysosomal Storage Diseases

Bev Davidson

October 2, 2007

Required Reading:

Sands MS, Davidson BL. Gene therapy for lysosomal storage diseases. Mol Ther. 2006 May;13(5):839-49. Epub 2006 Mar 20. Review.

Suggested, relevant papers:

Jeyakumar M, Dwek RA, Butters TD, Platt FM. Storage solutions: treating lysosomal disorders of the brain. Nat Rev Neurosci. 2005 Sep;6(9):713-25.

Kumar P, Wu H, McBride JL, Jung KE, Kim MH, Davidson BL, Lee SK, Shankar P, Manjunath N. Transvascular delivery of small interfering RNA to the central nervous system. Nature. 2007 Jul 5;448(7149):39-43.

PowerPoint Presentation:

Lecture provided in class