Gene therapy brings new hope for Mucopolysaccharidosis Type I patients

Gene therapy brings new hope for Mucopolysaccharidosis Type I patients

Dhaka the 24th October, 2014. Mucopolysaccharidosis Type I (MPS I) is a genetic diseases resulting from insufficiency of a lysosomal enzyme, α-L-iduronidase (IDUA). This enzyme plays an important role in the breakdown of glycoseaminoglycan (GAG).  The absence or reduced activity of this enzyme leads to accumulation of heparan sulfate and dermatan sulfate in different tissues causing  several diseases.

Hematopoetic Stem Cell transplantation or regular enzyme infusion are the only treatment we have till date but these often compromise with the  safety of the patient and in several cases leading to critical clinical  conditions. Recently, some researchers from three different institutes led by James M. Wilson designed a gene therapy (doi:10.1073/pnas.1413645111); they constructed a virus based vector for IDUA gene reconstitution targeting the liver. After applying the gene therapy to  3-4 months old MPS I cat models they screened them for 6 months. Three of the four cats produced effective IDUA enzyme more than the normal.  Only one produced 30% compared to the  normal IDUA level. In addition, the models showed reduced deposition of  GAGs and most importantly, the clinical phenomena like aortic valve lesions are also observed here. The results could initiate the new hope for safer treatment of MPS I in human. [Summarized by  Ahsan Polash]

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