Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I
Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM. Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I. Proc Natl Acad Sci U S A. 2014 Sep 29. pii: 201413645. [Epub ahead of print] | doi:10.1073/pnas.1413645111
In the second of two papers outlining new gene-therapy approaches to treat a rare disease called MPS I, the Wilson Lab examined systemic delivery of a vector to replace the enzyme IDUA, which is deficient in patients with this disorder. MPS I eventually leads to the abnormal accumulation of certain molecules and cell death. The second paper, published in the Proceedings of the National Academy of Sciences, describes how an injection of a vector expressing the IDUA enzyme to the liver can prevent most of the systemic manifestations of the disease, including those found in the heart. The first paper, published in Molecular Therapy, describes the use of an adeno-associated viral (AAV) vector to introduce normal IDUA to glial and neuronal cells in the brain and spinal cord in a feline model. The aim of that study was to directly treat the central nervous system manifestations of MPS while the more recent study aims to treat all other manifestations of the disease outside of the nervous system.
Read the Department of Communications news release.