Assessment of TAT-PTD in the transduction of cystatin B for protein replacement therapy in Unverricht-Lundborg progressive myoclonous epilepsy.
Published: 2004
Language: eng
Number of pages: 100
Description: Unverricht-Lundborg disease (ULD) is the most common form of Progressive Myoclonus Epilepsy and is inherited in an autosomal recessive manner. Despite partial seizure control obtained with antiepileptic drugs, patients eventually develop dementia, ataxia, and tremor and after 10--20 years they are incapacitated. The gene responsible for ULD is called EPM1 and encodes for the cysteine proteinase inhibitor cystatin B (CSTB). The identification of EPM1 has provided opportunity for the development and testing of new therapies.Protein transduction domains (PTDs) were initially thought capable of transducing proteins attached to them across plasma cell membranes and into the central nervous system. These characteristics prompted me to establish a TAT-PTD-CSTB delivery system using a cell-based assay to develop protein-replacement therapies for ULD. My data demonstrated that no transduction occurred for CSTB, and therefore, this system is not suitable for therapy of ULD.
Notes: Adviser:  Stephen W. Scherer.

Thesis (M.Sc.)--University of Toronto, 2004.

Electronic version licensed for access by U. of T. users.

Source: Masters Abstracts International, Volume: 42-06, page: 2111.

MICR copy on microfiche (2 microfiches).