Stéphanie Astord

Research Assistant

Stéphanie Astord

Titles and positions

Research Assistant  (AIM)


  • AAV Production
  • Molecular and Cellular Biology, Biochemistry (western blotting and immunohistochemistry)


Graduated with a professional Master Degree in Genetics, Physiology and Biotechnologies in 2007.

Since 2007, I’m working as engineer in the team of Martine Barkats before and Maria Grazia Biferi after. This allowed me to acquire various knowledge and skills on motor neuron diseases and particularly ALS and SMA, but also to contribute to the conception of innovative gene therapies for these diseases. I am also involved in the production of recombinant AAV, essential vectors for the development of our therapeutic strategies.

Main publications

  1. Niemir, N, Rouvière, L, Besse, A, Vanier, MT, Dmytrus, J, Marais, T et al.. Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice. Hum Mol Genet. 2018;27 (6):954-968. doi: 10.1093/hmg/ddy012. PubMed PMID:29325092 .
  2. Puzzo, F, Colella, P, Biferi, MG, Bali, D, Paulk, NK, Vidal, P et al.. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. Sci Transl Med. 2017;9 (418):. doi: 10.1126/scitranslmed.aam6375. PubMed PMID:29187643 PubMed Central PMC5826611.
  3. Biferi, MG, Cohen-Tannoudji, M, Cappelletto, A, Giroux, B, Roda, M, Astord, S et al.. A New AAV10-U7-Mediated Gene Therapy Prolongs Survival and Restores Function in an ALS Mouse Model. Mol Ther. 2017;25 (9):2038-2052. doi: 10.1016/j.ymthe.2017.05.017. PubMed PMID:28663100 PubMed Central PMC5589057.
  4. Tanguy, Y, Biferi, MG, Besse, A, Astord, S, Cohen-Tannoudji, M, Marais, T et al.. Systemic AAVrh10 provides higher transgene expression than AAV9 in the brain and the spinal cord of neonatal mice. Front Mol Neurosci. 2015;8 :36. doi: 10.3389/fnmol.2015.00036. PubMed PMID:26283910 PubMed Central PMC4516891.
  5. Bucher, T, Colle, MA, Wakeling, E, Dubreil, L, Fyfe, J, Briot-Nivard, D et al.. scAAV9 intracisternal delivery results in efficient gene transfer to the central nervous system of a feline model of motor neuron disease. Hum Gene Ther. 2013;24 (7):670-82. doi: 10.1089/hum.2012.218. PubMed PMID:23799774 .
  6. Bemelmans, AP, Duqué, S, Rivière, C, Astord, S, Desrosiers, M, Marais, T et al.. A single intravenous AAV9 injection mediates bilateral gene transfer to the adult mouse retina. PLoS One. 2013;8 (4):e61618. doi: 10.1371/journal.pone.0061618. PubMed PMID:23613884 PubMed Central PMC3626698.
  7. Benkhelifa-Ziyyat, S, Besse, A, Roda, M, Duque, S, Astord, S, Carcenac, R et al.. Intramuscular scAAV9-SMN injection mediates widespread gene delivery to the spinal cord and decreases disease severity in SMA mice. Mol Ther. 2013;21 (2):282-90. doi: 10.1038/mt.2012.261. PubMed PMID:23295949 PubMed Central PMC3594018.
  8. Blumen, SC, Astord, S, Robin, V, Vignaud, L, Toumi, N, Cieslik, A et al.. A rare recessive distal hereditary motor neuropathy with HSJ1 chaperone mutation. Ann Neurol. 2012;71 (4):509-19. doi: 10.1002/ana.22684. PubMed PMID:22522442 .
  9. Dominguez, E, Marais, T, Chatauret, N, Benkhelifa-Ziyyat, S, Duque, S, Ravassard, P et al.. Intravenous scAAV9 delivery of a codon-optimized SMN1 sequence rescues SMA mice. Hum Mol Genet. 2011;20 (4):681-93. doi: 10.1093/hmg/ddq514. PubMed PMID:21118896 .
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