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Items: 1 to 20 of 25


Delivery of Adeno-Associated Virus Gene Therapy by Intravascular Limb Infusion Methods.

Gruntman AM, Flotte TR.

Hum Gene Ther Clin Dev. 2015 Sep;26(3):159-64. doi: 10.1089/humc.2015.116. Epub 2015 Sep 10.


Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system.

Meliani A, Leborgne C, Triffault S, Jeanson-Leh L, Veron P, Mingozzi F.

Hum Gene Ther Methods. 2015 Apr;26(2):45-53. doi: 10.1089/hgtb.2015.037.


Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.

Nichols TC, Whitford MH, Arruda VR, Stedman HH, Kay MA, High KA.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):5-14. doi: 10.1089/humc.2014.153. Epub 2015 Feb 12. Review.


AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice.

Crudele JM, Finn JD, Siner JI, Martin NB, Niemeyer GP, Zhou S, Mingozzi F, Lothrop CD Jr, Arruda VR.

Blood. 2015 Mar 5;125(10):1553-61. doi: 10.1182/blood-2014-07-588194. Epub 2015 Jan 7.


Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

Le Guiner C, Montus M, Servais L, Cherel Y, Francois V, Thibaud JL, Wary C, Matot B, Larcher T, Guigand L, Dutilleul M, Domenger C, Allais M, Beuvin M, Moraux A, Le Duff J, Devaux M, Jaulin N, Guilbaud M, Latournerie V, Veron P, Boutin S, Leborgne C, Desgue D, Deschamps JY, Moullec S, Fromes Y, Vulin A, Smith RH, Laroudie N, Barnay-Toutain F, Rivière C, Bucher S, Le TH, Delaunay N, Gasmi M, Kotin RM, Bonne G, Adjali O, Masurier C, Hogrel JY, Carlier P, Moullier P, Voit T.

Mol Ther. 2014 Nov;22(11):1923-35. doi: 10.1038/mt.2014.151. Epub 2014 Aug 4.


Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions.

Basner-Tschakarjan E, Mingozzi F.

Front Immunol. 2014 Jul 23;5:350. doi: 10.3389/fimmu.2014.00350. eCollection 2014. Review.


Muscle Gene Therapy for Hemophilia.

Sabatino DE, Arruda VR.

J Genet Syndr Gene Ther. 2012 May 7;Suppl 1. pii: S1-010.


Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside.

Ferreira V, Petry H, Salmon F.

Front Immunol. 2014 Mar 3;5:82. doi: 10.3389/fimmu.2014.00082. eCollection 2014.


The potential of adeno-associated viral vectors for gene delivery to muscle tissue.

Wang D, Zhong L, Nahid MA, Gao G.

Expert Opin Drug Deliv. 2014 Mar;11(3):345-64. doi: 10.1517/17425247.2014.871258. Epub 2014 Jan 3. Review.


A novel cell-sheet technology that achieves durable factor VIII delivery in a mouse model of hemophilia A.

Tatsumi K, Sugimoto M, Lillicrap D, Shima M, Ohashi K, Okano T, Matsui H.

PLoS One. 2013 Dec 16;8(12):e83280. doi: 10.1371/journal.pone.0083280. eCollection 2013.


Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL(S447X)) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy.

Ferreira V, Twisk J, Kwikkers K, Aronica E, Brisson D, Methot J, Petry H, Gaudet D.

Hum Gene Ther. 2014 Mar;25(3):180-8. doi: 10.1089/hum.2013.169. Epub 2014 Feb 28.


Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia.

Chen SJ, Sanmiguel J, Lock M, McMenamin D, Draper C, Limberis MP, Kassim SH, Somanathan S, Bell P, Johnston JC, Rader DJ, Wilson JM.

Hum Gene Ther Clin Dev. 2013 Dec;24(4):154-60. doi: 10.1089/humc.2013.082. Epub 2013 Nov 9.


Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice.

Chen Y, Schroeder JA, Kuether EL, Zhang G, Shi Q.

Mol Ther. 2014 Jan;22(1):169-77. doi: 10.1038/mt.2013.197. Epub 2013 Aug 23.


Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX.

Shi Y, Falahati R, Zhang J, Flebbe-Rehwaldt L, Gaensler KM.

Gene Ther. 2013 Oct;20(10):987-96. doi: 10.1038/gt.2013.22. Epub 2013 Jun 13.


Immune responses to AAV vectors: overcoming barriers to successful gene therapy.

Mingozzi F, High KA.

Blood. 2013 Jul 4;122(1):23-36. doi: 10.1182/blood-2013-01-306647. Epub 2013 Apr 17. Review.


Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy.

Callejas D, Mann CJ, Ayuso E, Lage R, Grifoll I, Roca C, Andaluz A, Ruiz-de Gopegui R, Montané J, Muñoz S, Ferre T, Haurigot V, Zhou S, Ruberte J, Mingozzi F, High KA, Garcia F, Bosch F.

Diabetes. 2013 May;62(5):1718-29. doi: 10.2337/db12-1113. Epub 2013 Feb 1.


Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype.

Siner JI, Iacobelli NP, Sabatino DE, Ivanciu L, Zhou S, Poncz M, Camire RM, Arruda VR.

Blood. 2013 May 23;121(21):4396-403. doi: 10.1182/blood-2012-10-464164. Epub 2013 Jan 31.


The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

Finn JD, Nichols TC, Svoronos N, Merricks EP, Bellenger DA, Zhou S, Simioni P, High KA, Arruda VR.

Blood. 2012 Nov 29;120(23):4521-3. doi: 10.1182/blood-2012-06-440123. Epub 2012 Aug 23.

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