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Items: 28

1.

In Vivo Gene Therapy for Mucopolysaccharidosis Type III (Sanfilippo Syndrome): A New Treatment Horizon.

Marcó S, Haurigot V, Bosch F.

Hum Gene Ther. 2019 Oct;30(10):1211-1221. doi: 10.1089/hum.2019.217.

PMID:
31482754
2.

FGF21 gene therapy as treatment for obesity and insulin resistance.

Jimenez V, Jambrina C, Casana E, Sacristan V, Muñoz S, Darriba S, Rodó J, Mallol C, Garcia M, León X, Marcó S, Ribera A, Elias I, Casellas A, Grass I, Elias G, Ferré T, Motas S, Franckhauser S, Mulero F, Navarro M, Haurigot V, Ruberte J, Bosch F.

EMBO Mol Med. 2018 Aug;10(8). pii: e8791. doi: 10.15252/emmm.201708791.

3.

AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice.

Mallol C, Casana E, Jimenez V, Casellas A, Haurigot V, Jambrina C, Sacristan V, Morró M, Agudo J, Vilà L, Bosch F.

Mol Metab. 2017 May 17;6(7):664-680. doi: 10.1016/j.molmet.2017.05.007. eCollection 2017 Jul.

4.

Long-Term Efficacy and Safety of Insulin and Glucokinase Gene Therapy for Diabetes: 8-Year Follow-Up in Dogs.

Jaén ML, Vilà L, Elias I, Jimenez V, Rodó J, Maggioni L, Ruiz-de Gopegui R, Garcia M, Muñoz S, Callejas D, Ayuso E, Ferré T, Grifoll I, Andaluz A, Ruberte J, Haurigot V, Bosch F.

Mol Ther Methods Clin Dev. 2017 Apr 5;6:1-7. doi: 10.1016/j.omtm.2017.03.008. eCollection 2017 Sep 15.

5.

Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.

Roca C, Motas S, Marcó S, Ribera A, Sánchez V, Sánchez X, Bertolin J, León X, Pérez J, Garcia M, Villacampa P, Ruberte J, Pujol A, Haurigot V, Bosch F.

Hum Mol Genet. 2017 Apr 15;26(8):1535-1551. doi: 10.1093/hmg/ddx058.

PMID:
28334745
6.

CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome).

Motas S, Haurigot V, Garcia M, Marcó S, Ribera A, Roca C, Sánchez X, Sánchez V, Molas M, Bertolin J, Maggioni L, León X, Ruberte J, Bosch F.

JCI Insight. 2016 Jun 16;1(9):e86696. doi: 10.1172/jci.insight.86696.

7.

Progressive neurologic and somatic disease in a novel mouse model of human mucopolysaccharidosis type IIIC.

Marcó S, Pujol A, Roca C, Motas S, Ribera A, Garcia M, Molas M, Villacampa P, Melia CS, Sánchez V, Sánchez X, Bertolin J, Ruberte J, Haurigot V, Bosch F.

Dis Model Mech. 2016 Sep 1;9(9):999-1013. doi: 10.1242/dmm.025171. Epub 2016 Aug 4.

8.

Insulin-like Growth Factor 2 Overexpression Induces β-Cell Dysfunction and Increases Beta-cell Susceptibility to Damage.

Casellas A, Mallol C, Salavert A, Jimenez V, Garcia M, Agudo J, Obach M, Haurigot V, Vilà L, Molas M, Lage R, Morró M, Casana E, Ruberte J, Bosch F.

J Biol Chem. 2015 Jul 3;290(27):16772-85. doi: 10.1074/jbc.M115.642041. Epub 2015 May 13.

9.

Proliferative retinopathies: animal models and therapeutic opportunities.

Villacampa P, Haurigot V, Bosch F.

Curr Neurovasc Res. 2015;12(2):189-98. Review.

PMID:
25760215
10.

Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy.

Ribera A, Haurigot V, Garcia M, Marcó S, Motas S, Villacampa P, Maggioni L, León X, Molas M, Sánchez V, Muñoz S, Leborgne C, Moll X, Pumarola M, Mingozzi F, Ruberte J, Añor S, Bosch F.

Hum Mol Genet. 2015 Apr 1;24(7):2078-95. doi: 10.1093/hmg/ddu727. Epub 2014 Dec 18.

PMID:
25524704
11.

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.

12.

Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy.

Haurigot V, Marcó S, Ribera A, Garcia M, Ruzo A, Villacampa P, Ayuso E, Añor S, Andaluz A, Pineda M, García-Fructuoso G, Molas M, Maggioni L, Muñoz S, Motas S, Ruberte J, Mingozzi F, Pumarola M, Bosch F.

J Clin Invest. 2013 Jul 1. pii: 66778. doi: 10.1172/JCI66778. [Epub ahead of print]

13.

Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes.

Hui DJ, Basner-Tschakarjan E, Chen Y, Davidson RJ, Buchlis G, Yazicioglu M, Pien GC, Finn JD, Haurigot V, Tai A, Scott DW, Cousens LP, Zhou S, De Groot AS, Mingozzi F.

Mol Ther. 2013 Sep;21(9):1727-37. doi: 10.1038/mt.2013.166. Epub 2013 Jul 16.

14.

Insulin-like growth factor I (IGF-I)-induced chronic gliosis and retinal stress lead to neurodegeneration in a mouse model of retinopathy.

Villacampa P, Ribera A, Motas S, Ramírez L, García M, de la Villa P, Haurigot V, Bosch F.

J Biol Chem. 2013 Jun 14;288(24):17631-42. doi: 10.1074/jbc.M113.468819. Epub 2013 Apr 25.

15.

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.

16.

Toward a gene therapy for neurological and somatic MPSIIIA.

Haurigot V, Bosch F.

Rare Dis. 2013 Dec 12;1:e27209. doi: 10.4161/rdis.27209. eCollection 2013.

17.

Long-term retinal PEDF overexpression prevents neovascularization in a murine adult model of retinopathy.

Haurigot V, Villacampa P, Ribera A, Bosch A, Ramos D, Ruberte J, Bosch F.

PLoS One. 2012;7(7):e41511. doi: 10.1371/journal.pone.0041511. Epub 2012 Jul 20.

18.

Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer.

Ruzo A, Marcó S, García M, Villacampa P, Ribera A, Ayuso E, Maggioni L, Mingozzi F, Haurigot V, Bosch F.

Hum Gene Ther. 2012 Dec;23(12):1237-46. doi: 10.1089/hum.2012.029. Epub 2012 Oct 17.

PMID:
22909060
19.

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.

20.

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.

21.

Inhibition of hepatitis C virus replication using adeno-associated virus vector delivery of an exogenous anti-hepatitis C virus microRNA cluster.

Yang X, Haurigot V, Zhou S, Luo G, Couto LB.

Hepatology. 2010 Dec;52(6):1877-87. doi: 10.1002/hep.23908. Epub 2010 Oct 7.

PMID:
20931557
22.

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

Haurigot V, Mingozzi F, Buchlis G, Hui DJ, Chen Y, Basner-Tschakarjan E, Arruda VR, Radu A, Franck HG, Wright JF, Zhou S, Stedman HH, Bellinger DA, Nichols TC, High KA.

Mol Ther. 2010 Jul;18(7):1318-29. doi: 10.1038/mt.2010.73. Epub 2010 Apr 27.

23.

Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.

Arruda VR, Stedman HH, Haurigot V, Buchlis G, Baila S, Favaro P, Chen Y, Franck HG, Zhou S, Wright JF, Couto LB, Jiang H, Pierce GF, Bellinger DA, Mingozzi F, Nichols TC, High KA.

Blood. 2010 Jun 10;115(23):4678-88. doi: 10.1182/blood-2009-12-261156. Epub 2010 Mar 24.

24.

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.

PMID:
19956269
25.

Increased intraocular insulin-like growth factor-I triggers blood-retinal barrier breakdown.

Haurigot V, Villacampa P, Ribera A, Llombart C, Bosch A, Nacher V, Ramos D, Ayuso E, Segovia JC, Bueren JA, Ruberte J, Bosch F.

J Biol Chem. 2009 Aug 21;284(34):22961-9. doi: 10.1074/jbc.M109.014787. Epub 2009 May 27.

26.

IGF-I mediates regeneration of endocrine pancreas by increasing beta cell replication through cell cycle protein modulation in mice.

Agudo J, Ayuso E, Jimenez V, Salavert A, Casellas A, Tafuro S, Haurigot V, Ruberte J, Segovia JC, Bueren J, Bosch F.

Diabetologia. 2008 Oct;51(10):1862-72. doi: 10.1007/s00125-008-1087-8. Epub 2008 Jul 29.

PMID:
18663428
27.

In vivo gene transfer to pancreatic beta cells by systemic delivery of adenoviral vectors.

Ayuso E, Chillón M, Agudo J, Haurigot V, Bosch A, Carretero A, Otaegui PJ, Bosch F.

Hum Gene Ther. 2004 Aug;15(8):805-12.

PMID:
15319037
28.

Increased ocular levels of IGF-1 in transgenic mice lead to diabetes-like eye disease.

Ruberte J, Ayuso E, Navarro M, Carretero A, Nacher V, Haurigot V, George M, Llombart C, Casellas A, Costa C, Bosch A, Bosch F.

J Clin Invest. 2004 Apr;113(8):1149-57.

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