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Items: 10


Patient-Specific iPSC-Derived Astrocytes Contribute to Non-Cell-Autonomous Neurodegeneration in Parkinson's Disease.

di Domenico A, Carola G, Calatayud C, Pons-Espinal M, Muñoz JP, Richaud-Patin Y, Fernandez-Carasa I, Gut M, Faella A, Parameswaran J, Soriano J, Ferrer I, Tolosa E, Zorzano A, Cuervo AM, Raya A, Consiglio A.

Stem Cell Reports. 2019 Feb 12;12(2):213-229. doi: 10.1016/j.stemcr.2018.12.011. Epub 2019 Jan 10.


Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII.

Marcos-Contreras OA, Smith SM, Bellinger DA, Raymer RA, Merricks E, Faella A, Pavani G, Zhou S, Nichols TC, High KA, Margaritis P.

Blood. 2016 Feb 4;127(5):565-71. doi: 10.1182/blood-2015-09-671420. Epub 2015 Dec 23.


The endothelial protein C receptor enhances hemostasis of FVIIa administration in hemophilic mice in vivo.

Pavani G, Ivanciu L, Faella A, Marcos-Contreras OA, Margaritis P.

Blood. 2014 Aug 14;124(7):1157-65. doi: 10.1182/blood-2014-04-567297. Epub 2014 Jun 23.


An engineered U1 small nuclear RNA rescues splicing defective coagulation F7 gene expression in mice.

Balestra D, Faella A, Margaritis P, Cavallari N, Pagani F, Bernardi F, Arruda VR, Pinotti M.

J Thromb Haemost. 2014 Feb;12(2):177-85. doi: 10.1111/jth.12471.


Overcoming preexisting humoral immunity to AAV using capsid decoys.

Mingozzi F, Anguela XM, Pavani G, Chen Y, Davidson RJ, Hui DJ, Yazicioglu M, Elkouby L, Hinderer CJ, Faella A, Howard C, Tai A, Podsakoff GM, Zhou S, Basner-Tschakarjan E, Wright JF, High KA.

Sci Transl Med. 2013 Jul 17;5(194):194ra92. doi: 10.1126/scitranslmed.3005795.


Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo.

Margaritis P, Roy E, Faella A, Downey HD, Ivanciu L, Pavani G, Zhou S, Bunte RM, High KA.

Blood. 2011 Apr 14;117(15):3974-82. doi: 10.1182/blood-2010-09-309732. Epub 2011 Feb 16. Erratum in: Blood. 2012 May 10;119(19):4577.


Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.

Cotugno G, Annunziata P, Tessitore A, O'Malley T, Capalbo A, Faella A, Bartolomeo R, O'Donnell P, Wang P, Russo F, Sleeper MM, Knox VW, Fernandez S, Levanduski L, Hopwood J, De Leonibus E, Haskins M, Auricchio A.

Mol Ther. 2011 Mar;19(3):461-9. doi: 10.1038/mt.2010.257. Epub 2010 Nov 30.


Noninvasive repetitive imaging of somatostatin receptor 2 gene transfer with positron emission tomography.

Cotugno G, Aurilio M, Annunziata P, Capalbo A, Faella A, Rinaldi V, Strisciuglio C, Di Tommaso M, Aloj L, Auricchio A.

Hum Gene Ther. 2011 Feb;22(2):189-96. doi: 10.1089/hum.2010.098. Epub 2011 Jan 11.


Different serum enzyme levels are required to rescue the various systemic features of the mucopolysaccharidoses.

Cotugno G, Tessitore A, Capalbo A, Annunziata P, Strisciuglio C, Faella A, Aurilio M, Di Tommaso M, Russo F, Mancini A, De Leonibus E, Aloj L, Auricchio A.

Hum Gene Ther. 2010 May;21(5):555-69. doi: 10.1089/hum.2009.189.


Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle.

Tessitore A, Faella A, O'Malley T, Cotugno G, Doria M, Kunieda T, Matarese G, Haskins M, Auricchio A.

Mol Ther. 2008 Jan;16(1):30-7. Epub 2007 Oct 23.

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