Mucopolysaccharidosis type IIIC (MPSIIIC) is a severe neurodegenerative lysosomal storage disease caused by loss-of-function of the lysosomal transmembrane protein heparan-alpha-glucosamine N-acetyltransferase (HGSNAT).

In this webinar, Rafael Badell-Grau, PhD, a post-doctoral fellow at the Cherqui lab, University of California, San Diego, will describe how the lab developed an ex vivo gene therapy using gene-modified hematopoietic stem and progenitor cells (HSPCs). These studies represent the first step towards clinical translation of an ex vivo gene therapy approach for MPSIIIC.

The speaker will present promising preclinical data and share valuable insights for those developing gene therapies for rare diseases, including details on developing a suitable mouse model, transduction of mice and patient cells lines using a self-inactivated lentivirus vector, and in vitro and in vivo testing.

Attend this webinar to:

• Explore the generation of novel mouse model for genetic diseases
• Understand the path to clinical application of ex vivo gene therapies
• Gain insight into the potential of HSPC transplantation for treating lysosomal storage disease
• Understand the impact of rare lysosomal disorder