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Cavazzana M. Miccio A., Andre-Schmutz I., and Mavilio F. Gene replacement therapy for hemoglobinopathies: clinical benefit and challenges for widespread utilization. Cell and Gene Therapy Insights 2018(7) 653-664. doi: 10.18609/cgti.2018.063


Moiani A., Paleari Y., Sartori D., Mezzadra R., Miccio A., Cattoglio C, Cocchiarella F., Lidonnici M.R., Ferrari G., and Mavilio, F. Integration of lentiviral vectors in the human genome induces alternative splicing and the generation of aberrant transcripts. ( Highlighted in: Trono, D. (2012) Gene therapy: too much splice can spoil the dish. J Clin Invest 122, 1600-1602.) J Clin Invest. 2012 May 1;122(5):1653-66.


Antoniani C. Meneghini V., Lattanzi A., Felix T., Romano O., Magrin E., Weber L., Pavani G., Nakamura Y., Cradick T.J., Lundberg A.S., Amendola M., El-Nemer W., Cavazzana M., Mavilio F. and Miccio A. Induction of fetal hemoglobin synthesis in human erythroblasts by CRISPR/Cas9-mediated editing of the β-globin locus. (Comment on the article by Montbleau KE and Sankaran VG: Blood. 2018 Apr 26;131(17):1884-1885. doi: 10.1182/blood-2018-03-839787) Blood. 2018 Mar 8. pii: blood-2017-10-811505. doi: 10.1182/blood-2017-10-811505. F1000Prime recommended.


Bonanomi D, Menegon A, Miccio A., Ferrari G, Corradi A, Kao HT, Benfenati F, Valtorta F. Phosphorylation of synapsin I by cAMP-dependent protein kinase controls synaptic vesicle dynamics in developing neurons. J Neurosci. 2005 Aug 10;25(32):7299-308.


Ferretti E, Villaescusa JC, Di Rosa P, Fernandez-Diaz LC, Longobardi E, Mazzieri R., Miccio A. Micali N, Selleri L, Ferrari G, Blasi F. Mutation of the TALE Gene Prep1 (pKnox1) Causes a Major Reduction of Pbx and Meis Proteins and a Pleiotropic Embryonic Phenotype. Mol Cell Biol. 2006 Aug;26(15):5650-62.


Cattoglio C, Facchini G, Sartori D, Antonelli A, Miccio A, Cassani B, Schmidt M, von Kalle C, Howe S, Thrasher AJ, Aiuti A, Ferrari G, Recchia A, Mavilio F. Hot spots of retroviral integration in human CD34+ hematopoietic cells. Blood 2007 Sep 15;110(6):1770-8.


Di Rosa P, Villaescusa JC, Longobardi E, Iotti G, Ferretti E, Diaz VM, Miccio A, Ferrari G, Blasi F. The homeodomain transcription factor Prep1 (pKnox1) is required for hematopoietic stem and progenitor cell activity. Dev Biol. 2007 Nov 15;311(2):324-34.


Miccio A, Cesari R, Lotti F, Rossi C, Sanvito F, Ponzoni M, Routledge SJ, Chow CM, Antoniou MN, Ferrari G. Miccio A, In vivo selection of genetically modified erythroblastic progenitors leads to long-term correction of beta-thalassemia. Proc Natl Acad Sci U S A. 2008 Jul 29;105(30):10547-52


Felice B. Cattoglio C., Cittaro D., Testa A., Miccio A., Ferrari G., Luzi L., Recchia A. and Mavilio F. Transcription factor binding sites as genetic determinants of retroviral integration in the human genome PLoS One. 2009;4(2):e4571. Epub 2009 Feb 24.


Gregory G.D.*, Miccio A.* Bersenev A., Wang Y., Hong W., Zhang Z., Poncz M., Tong W., Blobel G.A. FOG1 requires NuRD to promote hematopoiesis and maintain lineage fidelity within the megakaryocytic–erythroid compartment Blood 2010 Mar 18;115(11): 2156-66. Epub 2010 Jan 11.


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