Ten plus one challenges in diseases of the lysosomal system

Gregory A. Grabowski, Chester Whitley

Research output: Contribution to journalArticlepeer-review

4 Scopus citations


The advent of the first effective specific therapy for a lysosomal storage disease (LSDs), Gaucher disease type 1, by Roscoe O. Brady was foundational for development of additional treatments for this group of rare diseases. The past 26 years, since the approval of enzyme therapy for Gaucher disease type 1, have witnessed a burgeoning understanding of LSDs at genetic, molecular, biochemical, cell biologic, and clinical levels. Simultaneously, this expansion of knowledge has exposed our incomplete understanding of the individual pathophysiologies of LSDs as well as difficult challenges for improvement in therapy and therapeutic outcomes for afflicted individuals. Here, 10 such challenges/problems representing major impediments, which need to be overcome, to move forward toward the goals of more effective and complete therapies for these devastating diseases.

Original languageEnglish (US)
Pages (from-to)38-46
Number of pages9
JournalMolecular Genetics and Metabolism
Issue number1-2
StatePublished - Jan 1 2017


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