Thérapie génique

Oumeya Adjali
Durée : 1881000
00
Introduction
Adeno-Associated Virus and rAAV vectors
rAAV vectors hold great promise
rAAV vectors are nowadays used in muliiple clinical applications
rAAV vectors, skeletal muscle and (completed) clinical trials
rAAV's mode of delivery and DMD
Locoregional IV: Principle
Locoregional IV: advantages
LR delivery of rAAV-GFP allows persisting GFP expression
DLD and rAAV gene therapy
Medicinal product to test : rAAV8-µDys
rAAV8-µDys (cMD1) in GRMD dogs - Locoregional injection
DMD and locoregional injection of rAAV8
Evaluation in GRMD dogs - Systemic treatment
Intermediate muscular biopsies
Clinical monitoring
Film : 6 month-old GRMD dog
Absence of cellular immune response against µDys
Take Home Message...
Equipes
Couverture médiatique
Questions - réponses
  • Introduction (0)
  • Adeno-Associated Virus and rAAV vectors (28000)
  • rAAV vectors hold great promise (121000)
  • rAAV vectors are nowadays used in muliiple clinical applications (180000)
  • rAAV vectors, skeletal muscle and (completed) clinical trials (237000)
  • rAAV's mode of delivery and DMD (290000)
  • Locoregional IV: Principle (354000)
  • Locoregional IV: advantages (416000)
  • LR delivery of rAAV-GFP allows persisting GFP expression (478000)
  • DLD and rAAV gene therapy (526000)
  • Medicinal product to test : rAAV8-µDys (608000)
  • rAAV8-µDys (cMD1) in GRMD dogs - Locoregional injection (663000)
  • DMD and locoregional injection of rAAV8 (779000)
  • Evaluation in GRMD dogs - Systemic treatment (860000)
  • Intermediate muscular biopsies (900000)
  • Clinical monitoring (997000)
  • Film : 6 month-old GRMD dog (1079000)
  • Absence of cellular immune response against µDys (1232000)
  • Take Home Message... (1292000)
  • Equipes (1379000)
  • Couverture médiatique (1420000)
  • Questions - réponses (1460000)
Oumeya Adjali

...