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Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.
Lhériteau E, Petit L, Weber M, Le Meur G, Deschamps JY, Libeau L, Mendes-Madeira A, Guihal C, François A, Guyon R, Provost N, Lemoine F, Papal S, El-Amraoui A, Colle MA, Moullier P, Rolling F. Lhériteau E, et al. Among authors: colle ma. Mol Ther. 2014 Feb;22(2):265-277. doi: 10.1038/mt.2013.232. Epub 2013 Oct 4. Mol Ther. 2014. PMID: 24091916 Free PMC article.
AAV-mediated Gene Therapy Halts Retinal Degeneration in PDE6β-deficient Dogs.
Pichard V, Provost N, Mendes-Madeira A, Libeau L, Hulin P, Tshilenge KT, Biget M, Ameline B, Deschamps JY, Weber M, Le Meur G, Colle MA, Moullier P, Rolling F. Pichard V, et al. Among authors: colle ma. Mol Ther. 2016 May;24(5):867-76. doi: 10.1038/mt.2016.37. Epub 2016 Feb 9. Mol Ther. 2016. PMID: 26857842 Free PMC article.
Vitrectomy Before Intravitreal Injection of AAV2/2 Vector Promotes Efficient Transduction of Retinal Ganglion Cells in Dogs and Nonhuman Primates.
Tshilenge KT, Ameline B, Weber M, Mendes-Madeira A, Nedellec S, Biget M, Provost N, Libeau L, Blouin V, Deschamps JY, Le Meur G, Colle MA, Moullier P, Pichard V, Rolling F. Tshilenge KT, et al. Among authors: colle ma. Hum Gene Ther Methods. 2016 Jun;27(3):122-34. doi: 10.1089/hgtb.2016.034. Hum Gene Ther Methods. 2016. PMID: 27229628
Intracerebral Gene Therapy Using AAVrh.10-hARSA Recombinant Vector to Treat Patients with Early-Onset Forms of Metachromatic Leukodystrophy: Preclinical Feasibility and Safety Assessments in Nonhuman Primates.
Zerah M, Piguet F, Colle MA, Raoul S, Deschamps JY, Deniaud J, Gautier B, Toulgoat F, Bieche I, Laurendeau I, Sondhi D, Souweidane MM, Cartier-Lacave N, Moullier P, Crystal RG, Roujeau T, Sevin C, Aubourg P. Zerah M, et al. Among authors: colle ma. Hum Gene Ther Clin Dev. 2015 Jun;26(2):113-24. doi: 10.1089/humc.2014.139. Epub 2015 Apr 28. Hum Gene Ther Clin Dev. 2015. PMID: 25758611 Clinical Trial.
55 results