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Items: 15


Oracle or false prophet? Can we predict AAV efficacy based on preexisting antibody titers?

Anguela XM, High KA.

Res Pract Thromb Haemost. 2019 Feb 28;3(2):149-151. doi: 10.1002/rth2.12190. eCollection 2019 Apr. No abstract available.


Entering the Modern Era of Gene Therapy.

Anguela XM, High KA.

Annu Rev Med. 2019 Jan 27;70:273-288. doi: 10.1146/annurev-med-012017-043332. Epub 2018 Nov 26.


Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA.

N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.


An edible switch for gene therapy.

Anguela XM, High KA.

Nat Biotechnol. 2016 Aug 9;34(8):824-5. doi: 10.1038/nbt.3645. No abstract available.


Adeno-associated viral vectors for the treatment of hemophilia.

High KA, Anguela XM.

Hum Mol Genet. 2016 Apr 15;25(R1):R36-41. doi: 10.1093/hmg/ddv475. Epub 2015 Nov 27. Review.


In vivo genome editing of the albumin locus as a platform for protein replacement therapy.

Sharma R, Anguela XM, Doyon Y, Wechsler T, DeKelver RC, Sproul S, Paschon DE, Miller JC, Davidson RJ, Shivak D, Zhou S, Rieders J, Gregory PD, Holmes MC, Rebar EJ, High KA.

Blood. 2015 Oct 8;126(15):1777-84. doi: 10.1182/blood-2014-12-615492. Epub 2015 Aug 21.


Robust ZFN-mediated genome editing in adult hemophilic mice.

Anguela XM, Sharma R, Doyon Y, Miller JC, Li H, Haurigot V, Rohde ME, Wong SY, Davidson RJ, Zhou S, Gregory PD, Holmes MC, High KA.

Blood. 2013 Nov 7;122(19):3283-7. doi: 10.1182/blood-2013-04-497354. Epub 2013 Oct 1.


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.


Nonviral-mediated hepatic expression of IGF-I increases Treg levels and suppresses autoimmune diabetes in mice.

Anguela XM, Tafuro S, Roca C, Callejas D, Agudo J, Obach M, Ribera A, Ruzo A, Mann CJ, Casellas A, Bosch F.

Diabetes. 2013 Feb;62(2):551-60. doi: 10.2337/db11-1776. Epub 2012 Oct 25.


Molecular signature of the immune and tissue response to non-coding plasmid DNA in skeletal muscle after electrotransfer.

Mann CJ, Anguela XM, Montané J, Obach M, Roca C, Ruzo A, Otaegui P, Mir LM, Bosch F.

Gene Ther. 2012 Dec;19(12):1177-86. doi: 10.1038/gt.2011.198. Epub 2011 Dec 15.


Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice.

Ruzo A, Garcia M, Ribera A, Villacampa P, Haurigot V, Marcó S, Ayuso E, Anguela XM, Roca C, Agudo J, Ramos D, Ruberte J, Bosch F.

Mol Ther. 2012 Feb;20(2):254-66. doi: 10.1038/mt.2011.220. Epub 2011 Oct 18.


In vivo genome editing restores haemostasis in a mouse model of haemophilia.

Li H, Haurigot V, Doyon Y, Li T, Wong SY, Bhagwat AS, Malani N, Anguela XM, Sharma R, Ivanciu L, Murphy SL, Finn JD, Khazi FR, Zhou S, Paschon DE, Rebar EJ, Bushman FD, Gregory PD, Holmes MC, High KA.

Nature. 2011 Jun 26;475(7355):217-21. doi: 10.1038/nature10177.


Skeletal muscle metabolism in the pathology and treatment of type 1 diabetes.

Mann CJ, Ayuso E, Anguela XM, Bosch F.

Curr Pharm Des. 2010;16(8):1002-20. Review.


High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency.

Ayuso E, Mingozzi F, Montane J, Leon X, Anguela XM, Haurigot V, Edmonson SA, Africa L, Zhou S, High KA, Bosch F, Wright JF.

Gene Ther. 2010 Apr;17(4):503-10. doi: 10.1038/gt.2009.157. Epub 2009 Dec 3.


Reversal of type 1 diabetes by engineering a glucose sensor in skeletal muscle.

Mas A, Montané J, Anguela XM, Muñoz S, Douar AM, Riu E, Otaegui P, Bosch F.

Diabetes. 2006 Jun;55(6):1546-53.

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