Biblio

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2024
Castiello MCarmina, Brandas C, Ferrari S, Porcellini S, Sacchetti N, Canarutto D, Draghici E, Merelli I, Barcella M, Pelosi G, et al. Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency. Sci Transl Med. 2024;16(733):eadh8162.
2023
Asperti C, Canarutto D, Porcellini S, Sanvito F, Cecere F, Vavassori V, Ferrari S, Rovelli E, Albano L, Jacob A, et al. Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated and . Mol Ther Methods Clin Dev. 2023;30:546-557.
2022
Ferrari S, Jacob A, Cesana D, Laugel M, Beretta S, Varesi A, Unali G, Conti A, Canarutto D, Albano L, et al. Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells. Cell Stem Cell. 2022;29(10):1428-1444.e9.
Capo V, Abinun M, Villa A. Osteoclast rich osteopetrosis due to defects in TCIRG1 gene. Bone. 2022:116519.
2020
Rigoni R, Fontana E, Dobbs K, Marrella V, Taverniti V, Maina V, Facoetti A, D'Amico G, Al-Herz W, Cruz-Munoz MErnesto, et al. Cutaneous barrier leakage and gut inflammation drive skin disease in Omenn Syndrome. J Allergy Clin Immunol. 2020.
2019
Bortolomai I, Sandri M, Draghici E, Fontana E, Campodoni E, Marcovecchio GEnza, Ferrua F, Perani L, Spinelli A, Canu T, et al. Gene Modification and Three-Dimensional Scaffolds as Novel Tools to Allow the Use of Postnatal Thymic Epithelial Cells for Thymus Regeneration Approaches. Stem Cells Transl Med. 2019.
Lanzi G, Ferraro RMonica, Masneri S, Piovani G, Barisani C, Sobacchi C, Villa A, Vezzoni P, Giliani S. Generation of 3 clones of induced pluripotent stem cells (iPSCs) from a patient affected by Autosomal Recessive Osteopetrosis due to mutations in TCIRG1 gene. Stem Cell Res. 2019;42:101660.
Ferrua F, Cicalese MPia, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo MEster, Calbi V, Assanelli AAngelo, et al. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. Lancet Haematol. 2019.
2018
Menale C, Campodoni E, Palagano E, Mantero S, Erreni M, Inforzato A, Fontana E, Schena F, Hof RVan't, Sandri M, et al. MSC-Seeded Biomimetic Scaffolds as a Factory of Soluble RANKL in Rankl-Deficient Osteopetrosis. Stem Cells Transl Med. 2018.
2016
Meneghini V, Frati G, Sala D, De Cicco S, Luciani M, Cavazzin C, Paulis M, Mentzen W, Morena F, Giannelli S, et al. Generation of Human Induced Pluripotent Stem Cell-Derived Bona Fide Neural Stem Cells for Ex Vivo Gene Therapy of Metachromatic Leukodystrophy. Stem Cells Transl Med. 2016.
Rigoni R, Grassi F, Villa A, Cassani B. RAGs and BUGS: an alliance for autoimmunity. Gut Microbes. 2016.
Palagano E, Susani L, Menale C, Ramenghi U, Berger M, Uva P, Oppo M, Vezzoni P, Villa A, Sobacchi C. Synonymous Mutations Add a Layer of Complexity in the Diagnosis of Human Osteopetrosis. J Bone Miner Res. 2016.
2015
Paulis M, Castelli A, Susani L, Lizier M, Lagutina I, Focarelli MLuisa, Recordati C, Uva P, Faggioli F, Neri T, et al. Chromosome transplantation as a novel approach for correcting complex genomic disorders. Oncotarget. 2015.
Neri T, Muggeo S, Paulis M, Caldana MElena, Crisafulli L, Strina D, Focarelli MLuisa, Faggioli F, Recordati C, Scaramuzza S, et al. Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts. Stem Cell Reports. 2015.
2013
Sobacchi C, Schulz A, Coxon FP, Villa A, Helfrich MH. Osteopetrosis: genetics, treatment and new insights into osteoclast function. Nat Rev Endocrinol. 2013.