source: American Society of Gene & Cell Therapy

year: 2020

Mucopolysaccharidoses (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.

MPS and Gene Therapy

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 VI, although it cannot reverse damage the disease has already caused. You may have heard that MPS I is also known as Hurler syndrome, while MPS II is referred to as Hunter syndrome and MPS III is called Sanfilippo syndrome.

Gene therapy introduces a working version of a gene into the cells in charge of creating key proteins or enzymes. These genetic instructions are delivered to the cells using a vector, or vehicle, which are often derived from viruses. Typically, viruses are used as vectors because they are capable of entering cells to deliver genetic material. Don’t worry. All viral genes are removed and the vector is modified to only deliver therapeutic genes to cells. With the right genetic instructions, cells can then produce functional enzymes that help break down further buildup of GAGs.

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