How Can Gene Therapy Help MPS Patients?

ASGCT Staff - October 29, 2019

There are seven different types of MPS, each caused by a different faulty gene. Gene therapy may be able to slow the progression of the MPS disorders I, II, III and IV, although it cannot reverse damage the disease has already caused. 

The American Society of Gene & Cell Therapy (ASGCT) released its latest resource profiling disease-specific treatment efforts, this time focusing on developing gene therapies for mucopolysaccharidoses (MPS).

The resources on MPS are now available on MPS are a group of genetic disorders that affect our body’s ability to break down and recycle sugar molecules called glycosaminoglycans, or GAGs, leading to buildup and causing severe symptoms. Gene therapy targets the root cause of disease and therefore may offer hope for slowing or halting the progression of some MPS disorders.

Watch ASGCT's Gene Therapy for MPS Video

The information on muscular dystrophy is part of the launch of a second round of material in ASGCT’s Patient Education program. ASGCT will release brand new resources weekly on the Patient Education portal

The full schedule for ASGCT’s new Patient Education resources to be released during the remainder of 2019, excepting one-week breaks for the ASGCT Policy Summit and Thanksgiving, is as follows:

  • Hemophilia— November 12
  • Pompe—November 19
  • CAR T Basics—December 3

Let us know what you think of this second batch of resources. Send feedback via the ASGCT Contact form or at

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