Rare Disease Report

Mouse Models for Sanfilippo Diseases Explained - In Detail

APRIL 04, 2013


Brian Bigger, PhD, leads the Stem Cell & Neurotherapies laboratory at the University of Manchester in Manchester U.K. The lab uses a multidisciplinary approach to investigate stem cell and gene therapies for neurological diseases, including Mucopolysaccharidosis (MPS) diseases such as MPS I (Hurler) and MPS IIIa (Sanfilippo). In this exclusive interview with Rare Disease Report, Dr. Bigger described in detail how his lab using our current understanding of the pathophysiology of each Sanfilippo type to test different treatment options for each. For more information about some of his lab’s work, click here

Two excellent articles describing the work being conducted in Dr. Bigger’s laboratory are available at PLOS and Nature

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