Exon skipping

Aurélie Goyenvalle
Durée : 1287000
00
Introduction
Therapeutic principle
Exon-skipping
Strategies to achieve exon-skipping
Limitations of AAV vectors for DMD treatment
Current tools for splice switching in DMD
The exon-skipping approach for DMD: where are we now ?
Tricyclo-DNA chemistry
TCDNA design for exon 23 skipping in mouse models of DMD
Pharmacokinetics of TcDNA in mdx
Comparison study between AON chemistries
Comparison study: Exon skipping and dystrophin levels
Comparison study: Exon skipping levels in CNS
Comparison study: functional rescue
Therapeutic evaluation of TcDNA in DKO mice
Long term effect of TcDNA treatment
TcDNA Efficacy and Tox profile
Short term serum profile
Long term serum biochemistry
Kidney and liver histopathology
Urinary profile - general biomarkers
Urinary profile - specific and early biomarkers
Conclusions and on going work
Acknowledgements
Collaborators and Contributors
Questions - réponses
  • Introduction (0)
  • Therapeutic principle (22000)
  • Exon-skipping (132000)
  • Strategies to achieve exon-skipping (166000)
  • Limitations of AAV vectors for DMD treatment (208000)
  • Current tools for splice switching in DMD (290000)
  • The exon-skipping approach for DMD: where are we now ? (312000)
  • Tricyclo-DNA chemistry (404000)
  • TCDNA design for exon 23 skipping in mouse models of DMD (450000)
  • Pharmacokinetics of TcDNA in mdx (490000)
  • Comparison study between AON chemistries (525000)
  • Comparison study: Exon skipping and dystrophin levels (560000)
  • Comparison study: Exon skipping levels in CNS (602000)
  • Comparison study: functional rescue (632000)
  • Therapeutic evaluation of TcDNA in DKO mice (694000)
  • Long term effect of TcDNA treatment (771000)
  • TcDNA Efficacy and Tox profile (808000)
  • Short term serum profile (865000)
  • Long term serum biochemistry (901000)
  • Kidney and liver histopathology (911000)
  • Urinary profile - general biomarkers (958000)
  • Urinary profile - specific and early biomarkers (971000)
  • Conclusions and on going work (1025000)
  • Acknowledgements (1092000)
  • Collaborators and Contributors (1100000)
  • Questions - réponses (1113000)
Aurélie Goyenvalle

...