Trusted Resources: Education
Scientific literature and patient education texts
Mucopolysaccharidoses (MPS) and Gene Therapy
source: American Society of Gene & Cell Therapy
year: 2020
summary/abstract: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.
read more
Related Content
-
Glaucoma in MucopolysaccharidosesMucopolysaccharidoses are a group of lys...
-
Europe Grants Orphan Drug Status to JR-441, ERT for Sanfilippo AThe European Commission has granted an o...
-
Diagnosis of Mucopolysaccharidoses and Mucolipidosis by Assaying Multiplex Enzymes and GlycosaminoglycansMucopolysaccharidoses (MPS) and mucolipi...
-
Walla Al-Hertani, MD, MS, FRCPC, FCCMG, FACMGDr. Walla Al-Hertani is a Medical Bioche...
-
UCSF – Lysosomal Storage Disease CenterThe UCSF Lysosomal Storage Disease Cente...
-
The Youngest Pair of Siblings With Mucopolysaccharidosis Type IVA to Receive Enzyme Replacement Therapy to Date: A C...Mucopolysaccharidosis type IVA (OMIM 253...
-
Enzyme Replacement Therapy With Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II: An Integrated Analysis o...Enzyme replacement therapy (ERT) improve...