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A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells.
Nat Biotechnol. 2021 Jan;39(1):47-55. doi: 10.1038/s41587-020-0741-7. Epub 2020 Nov 16.
Nat Biotechnol. 2021.
PMID: 33199875
Free PMC article.
Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy.
Finn JD, Ozelo MC, Sabatino DE, Franck HW, Merricks EP, Crudele JM, Zhou S, Kazazian HH, Lillicrap D, Nichols TC, Arruda VR.
Finn JD, et al.
Blood. 2010 Dec 23;116(26):5842-8. doi: 10.1182/blood-2010-06-288001. Epub 2010 Sep 28.
Blood. 2010.
PMID: 20876851
Free PMC article.
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Recombinant canine B-domain-deleted FVIII exhibits high specific activity and is safe in the canine hemophilia A model.
Sabatino DE, Freguia CF, Toso R, Santos A, Merricks EP, Kazazian HH Jr, Nichols TC, Camire RM, Arruda VR.
Sabatino DE, et al.
Blood. 2009 Nov 12;114(20):4562-5. doi: 10.1182/blood-2009-05-220327. Epub 2009 Sep 21.
Blood. 2009.
PMID: 19770361
Free PMC article.
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Efficacy and safety of long-term prophylaxis in severe hemophilia A dogs following liver gene therapy using AAV vectors.
Sabatino DE, Lange AM, Altynova ES, Sarkar R, Zhou S, Merricks EP, Franck HG, Nichols TC, Arruda VR, Kazazian HH Jr.
Sabatino DE, et al.
Mol Ther. 2011 Mar;19(3):442-9. doi: 10.1038/mt.2010.240. Epub 2010 Nov 16.
Mol Ther. 2011.
PMID: 21081906
Free PMC article.
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