Mavilio, Fulvio

Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

Le Guiner, C., Servais, L., Montus, M., Larcher, T., Fraysse, B., Moullec, S., Allais, M., François, V., Dutilleul, M., Malerba, A., et al. (2017) · Nature Communications

Virus-based gene therapy researchMuscle Physiology and Disorders

Cited by 246

Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus

Antoniani, C., Meneghini, V., Lattanzi, A., Felix, T., Romano, O., Magrin, E., Weber, L., Pavani, G., El Hoss, S., Kurita, R., et al. (2018) · Blood

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 194

Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy

Childers, M. K., Joubert, R., Poulard, K., Moal, C., Grange, R. W., Doering, J. A., Lawlor, M. W., Rider, B. E., Jamet, T., Danièle, N., et al. (2014) · Science Translational Medicine

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 173

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

Weber, L., Frati, G., Felix, T., Hardouin, G., Casini, A., Wollenschlaeger, C., Meneghini, V., Masson, C., De Cian, A., Chalumeau, A., et al. (2020) · Science Advances

Hemoglobinopathies and Related DisordersCRISPR and Genetic Engineering

Cited by 140

Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs

Mack, D. L., Poulard, K., Goddard, M. A., Latournerie, V., Snyder, J. M., Grange, R. W., Elverman, M. R., Denard, J., Veron, P., Buscara, L., et al. (2017) · Molecular Therapy

Virus-based gene therapy researchNeurogenetic and Muscular Disorders Research

Cited by 114

Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair

Izmiryan, A., Ganier, C., Bovolenta, M., Schmitt, A., Mavilio, F., & Hovnanian, A. (2018) · Molecular Therapy Nucleic Acids

CRISPR and Genetic EngineeringSkin and Cellular Biology Research

Cited by 68

Preclinical Corrective Gene Transfer in Xeroderma Pigmentosum Human Skin Stem Cells

Warrick, E., Garcia, M., Chagnoleau, C., Chevallier, O., Bergoglio, V., Sartori, D., Mavilio, F., Angulo, J. F., Avril, M.-F., Sarasin, A., et al. (2012) · Molecular Therapy

Virus-based gene therapy researchDNA Repair Mechanisms

Cited by 58

Risk assessment in skin gene therapy: viral–cellular fusion transcripts generated by proviral transcriptional read-through in keratinocytes transduced with self-inactivating lentiviral vectors

Almarza, D., Bussadori, G., Navarro, M., Mavilio, F., Larcher, F., & Murillas, R. (2011) · Gene Therapy

Virus-based gene therapy researchRNA Interference and Gene Delivery

Cited by 56

Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates.

Matet, A., Kostic, C., Bemelmans, A.-P., Moulin, A., Rosolen, S. G., Martin, S., Mavilio, F., Amirjanians, V., Stieger, K., Lorenz, B., et al. (2017) · Translational Research

Retinal Development and DisordersVirus-based gene therapy research

Cited by 20