Mucolipidosis research update – December 2016

What a great way to finish off the year. In my role as vice-President for research with ISMRD it has been a great pleasure to award a research grant to researchers in the US who will explore gene therapy in an animal model of Mucolipidosis alpha/beta Type II. It is a 2-year grant that will utilise a naturally occurring feline model of the disease to explore delivery of a good copy of the defective gene. Read more at this link.

The drive to fundraise commenced in the early part of 2016 and was very well supported by affected families and partner organisations, with US$150,000 raised. It is particularly satisfying to have run a successful fundraiser, issued a call for proposals, have them received and evaluated and a decision made, all before the close of the year.