RNA-based therapeutic approaches for FTDP-17

Kavitha, Siva (2015) RNA-based therapeutic approaches for FTDP-17. PhD thesis, University of Trento.

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Neurodegenerative diseases are linked to altered splicing mechanisms (Mills et al., 2012). Fronto temporal dementia and parkinsonism linked to chromosome 17 (FTDP-17) is one such disease that stems from the differential splicing caused due to mutations in Microtubule associated protein tau (MAPT) gene (Esther et al., 2002). This PhD thesis focuses on developing RNA-based therapeutic approaches to address FTDP-17. CHAPTER 1 introduces a broad range of topics such as splicing mechanism, neurodegenerative diseases associated with splice defects, therapeutic tools to modulate such splice defects in the context of neurogenetic diseases and possible applications of available tools for FTDP-17. CHAPTER 2 explores an exon skipping strategy to modulate splice defects in the context of FTD-17 using small nuclear RNAs (snRNAs). CHAPTER 3 is based on a short interfering RNA (siRNA) approach to modulate post-transcriptional gene silencing of specific isoform associated to FTDP-17. CHAPTER 4 employs long non coding RNA (lncRNA) to mediate post transcriptional repression of tau protein associated to FTDP-17 and deciphers its auxiliary role in splicing of exon 10 CHAPTER 5 elaborates on the future perspectives of all the above mentioned approaches to find a cure for FTDP-17.

Item Type:Doctoral Thesis (PhD)
Doctoral School:Biomolecular Sciences
PhD Cycle:XXVI
Subjects:Area 05 - Scienze biologiche > BIO/15 BIOLOGIA FARMACEUTICA
Repository Staff approval on:03 Jul 2015 12:57

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