Open Access
Numéro
Cah. Myol.
Numéro 23, juillet 2021
Page(s) 12 - 17
Section Mise au point
DOI https://doi.org/10.1051/myolog/202123004
Publié en ligne 2 août 2021
  1. Mendell JR, Al-Zaidy SA, Rodino-Klapac LR. Current clinical applications of in vivo gene therapy with AAVs. Mol Ther 2021 ; 29 : 464–88. [CrossRef] [Google Scholar]
  2. Louis Jeune V, Joergensen JA, Hajjar RJ, Weber T. Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy. Hum Gene Ther Methods 2013 ; 24 : 59–67. [CrossRef] [Google Scholar]
  3. McGreevy JW, Hakim CH, McIntosh MA, et al. Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy. Dis Model Mech 2015 ; 8 : 195–213. [CrossRef] [PubMed] [Google Scholar]
  4. Duan D. Systemic AAV micro-dystrophin gene therapy for Duchenne muscular dystrophy. Mol Ther 2018 ; 26 : 2337–56. [CrossRef] [Google Scholar]
  5. Harper SQ, Hauser MA, DelloRusso C, et al. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med 2002 ; 8 : 253–61. [CrossRef] [PubMed] [Google Scholar]
  6. Banks GB, Judge LM, Allen JM, et al. The polyproline site in hinge 2 influences the functional capacity of truncated dystrophins. PLoS Genet 2010 ; 6 : e1000958. [CrossRef] [Google Scholar]
  7. Shieh PB. Emerging strategies in the treatment of Duchenne muscular dystrophy. Neurotherapeutics 2018 ; 15 : 840–8. [CrossRef] [Google Scholar]
  8. Le Guiner C, Servais L, Montus M, et al. Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Nat Commun 2017 ; 8 : 16105. [CrossRef] [Google Scholar]
  9. Hayashita-Kinoh H, Guillermo PH, Nitahara-Kasahara Y, et al. Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy. Mol Ther Methods Clin Dev 2020 ; 20 : 122–41. [Google Scholar]
  10. Buscara L, Gross DA, Daniele N. Of rAAV et men: from genetic neuromuscular disorder efficacy and toxicity preclinical studies to clinical trials and back. J Pers Med 2020 ; 10 : 258. [CrossRef] [Google Scholar]
  11. Hakim CH, Wasala NB, Pan X, et al. A five-repeat microdystrophin gene ameliorated dystrophic phenotype in the severe dba/2j-mdx model of Duchenne muscular dystrophy. Mol Ther Methods Clin Dev 2017 ; 6 : 216–30. [CrossRef] [Google Scholar]
  12. Fortunato F, Rossi R, Falzarano MS, et al. Innovative therapeutic approaches for Duchenne muscular dystrophy. J Clin Med 2021 ; 10 : 820. [CrossRef] [Google Scholar]
  13. Mendell JR, Sahenk Z, Lehman K, et al. Assessment of systemic delivery of AAVrh74.MHCK7 micro-dystrophin in children with Duchenne muscular dystrophy: a nonrandomized controlled trial. JAMA Neurol 2020 ; 77 : 1129–31. [CrossRef] [Google Scholar]
  14. Pfizer’s new phase 1b results of gene therapy in ambulatory boys with Duchenne muscular dystrophy (DMD) support advancement into pivotal phase 3 studyPfizer - Communiqué de presse du 15 mai 2020. [Google Scholar]
  15. Luo S, Hu D, Wang M, et al. Complement in hemolysis- and thrombosis-related diseases. Front Immunol 2020 ; 11 : 1212. [CrossRef] [Google Scholar]
  16. Monteiro Pereira Palma L, Sridharan M, Sethi S. Complement in secondary thrombotic microangiopathy. Kidney Int Rep 2021 ; 6 : 11–23. [CrossRef] [Google Scholar]
  17. Lemaire M, Noone D, Lapeyraque AL, et al. Inherited kidney complement diseases. Clin J Am Soc Nephrol 2021 ; 16 : 942–56. [CrossRef] [Google Scholar]
  18. Chand DH, Zaidman C, Arya K, et al. Thrombotic microangiopathy following onasemnogene abeparvovec for spinal muscular atrophy: a case series. J Pediatr 2021 ; 231 : 265–8. [CrossRef] [Google Scholar]
  19. Li C, Goudy K, Hirsch, et al. Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc Natl Acad Sci USA 2009 ; 106 : 10770–4. [CrossRef] [Google Scholar]
  20. George LA. No CpGs for AAVs? Blood 2021 ; 137 : 721–3. [CrossRef] [Google Scholar]
  21. Mendell JR, Campbell K, Rodino-Klapac L, et al. Dystrophin immunity in Duchenne’s muscular dystrophy. N Engl J Med 2010 ; 363 : 1429–37. [CrossRef] [Google Scholar]

Les statistiques affichées correspondent au cumul d'une part des vues des résumés de l'article et d'autre part des vues et téléchargements de l'article plein-texte (PDF, Full-HTML, ePub... selon les formats disponibles) sur la platefome Vision4Press.

Les statistiques sont disponibles avec un délai de 48 à 96 heures et sont mises à jour quotidiennement en semaine.

Le chargement des statistiques peut être long.