Biodistribution of AAVpo1 and AAVpo1A1 in mice with myotubular myopathy - Approches génétiques intégrées et nouvelles thérapies pour les maladies rares Access content directly
Conference Poster Year : 2020

Biodistribution of AAVpo1 and AAVpo1A1 in mice with myotubular myopathy

Paloma Navas-Navarro
  • Function : Author
Jihad Elandari
  • Function : Author
Louise Mangin
  • Function : Author
Karine Poulard
  • Function : Author
Samia Martin
  • Function : Author
  • PersonId : 938057
Dirk Grimm
  • Function : Author
Ana Buj-Bello


Recombinant adeno-associated viral (AAV) vectors are powerful vehicles for in vivo delivery of therapeutic transgenes. Tissue tropism and consequently vector dosage for gene therapy can vary amongst naturally occurring and engineered AAV serotypes. We previously reported that serotype 8 AAV-mediated MTM1 gene delivery can correct the phenotype of murine and canine models of myotubular myopathy, a severe pediatric disorder leading to generalized muscle weakness and respiratory failure in patients. In order to identify AAV vectors with increased skeletal muscle potency in Mtm1 deficient muscle, in the present study we compared the biodistribution and transgene expression of serotype 8, 9 and rh10 vectors with AAVpo1, and its derivative AAVpo1A1, of porcine origin that carry a Mtm1 expression cassette. A single dose of 2x1013vg/kg of each vector was administrated intravenously in mutant mice and tissues were harvested 4 weeks postinjection. We found that administration of AAV9-, AAVrh10- and AAVpo1A1-Mtm1 vectors resulted in higher myotubularin expression levels in skeletal muscles compared to AAV8- and AAVpo1-Mtm1 vectors. In addition, the AAVpo1A1 vector detargeted liver transduction. These results indicate that AAVpo1A1 capsid might be suitable for gene transfer in XLMTM muscles.
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hal-04442079 , version 1 (06-02-2024)


  • HAL Id : hal-04442079 , version 1


Edith Renaud-Gabardos, Paloma Navas-Navarro, Jihad Elandari, Louise Mangin, Karine Poulard, et al.. Biodistribution of AAVpo1 and AAVpo1A1 in mice with myotubular myopathy. ASGCT 2020, May 2020, Boston (MA), United States. ⟨hal-04442079⟩
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