Format
Sort by
Items per page

Send to

Choose Destination

Search results

Items: 1 to 50 of 161

1.

Treatment of brain disease in the mucopolysaccharidoses.

Scarpa M, Orchard PJ, Schulz A, Dickson PI, Haskins ME, Escolar ML, Giugliani R.

Mol Genet Metab. 2017 Dec;122S:25-34. doi: 10.1016/j.ymgme.2017.10.007. Epub 2017 Oct 16. Review.

2.

Evaluation of Intrathecal Routes of Administration for Adeno-Associated Viral Vectors in Large Animals.

Hinderer C, Bell P, Katz N, Vite CH, Louboutin JP, Bote E, Yu H, Zhu Y, Casal ML, Bagel J, O'Donnell P, Wang P, Haskins ME, Goode T, Wilson JM.

Hum Gene Ther. 2018 Jan;29(1):15-24. doi: 10.1089/hum.2017.026. Epub 2017 Oct 3.

PMID:
28806897
3.

Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model.

Hinderer C, Bell P, Louboutin JP, Katz N, Zhu Y, Lin G, Choa R, Bagel J, O'Donnell P, Fitzgerald CA, Langan T, Wang P, Casal ML, Haskins ME, Wilson JM.

Mol Genet Metab. 2016 Sep;119(1-2):124-30. doi: 10.1016/j.ymgme.2016.06.006. Epub 2016 Jun 8.

4.

Pentosan Polysulfate: Oral Versus Subcutaneous Injection in Mucopolysaccharidosis Type I Dogs.

Simonaro CM, Tomatsu S, Sikora T, Kubaski F, Frohbergh M, Guevara JM, Wang RY, Vera M, Kang JL, Smith LJ, Schuchman EH, Haskins ME.

PLoS One. 2016 Apr 11;11(4):e0153136. doi: 10.1371/journal.pone.0153136. eCollection 2016.

5.

The influence of nasal airflow on respiratory and olfactory epithelial distribution in felids.

Pang B, Yee KK, Lischka FW, Rawson NE, Haskins ME, Wysocki CJ, Craven BA, Van Valkenburgh B.

J Exp Biol. 2016 Jun 15;219(Pt 12):1866-74. doi: 10.1242/jeb.131482. Epub 2016 Apr 4.

6.

Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.

Callan MB, Haskins ME, Wang P, Zhou S, High KA, Arruda VR.

PLoS One. 2016 Mar 24;11(3):e0151800. doi: 10.1371/journal.pone.0151800. eCollection 2016.

7.

Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII.

Gurda BL, De Guilhem De Lataillade A, Bell P, Zhu Y, Yu H, Wang P, Bagel J, Vite CH, Sikora T, Hinderer C, Calcedo R, Yox AD, Steet RA, Ruane T, O'Donnell P, Gao G, Wilson JM, Casal M, Ponder KP, Haskins ME.

Mol Ther. 2016 Feb;24(2):206-216. doi: 10.1038/mt.2015.189. Epub 2015 Oct 8.

8.

Delayed hypertrophic differentiation of epiphyseal chondrocytes contributes to failed secondary ossification in mucopolysaccharidosis VII dogs.

Peck SH, O'Donnell PJ, Kang JL, Malhotra NR, Dodge GR, Pacifici M, Shore EM, Haskins ME, Smith LJ.

Mol Genet Metab. 2015 Nov;116(3):195-203. doi: 10.1016/j.ymgme.2015.09.008. Epub 2015 Sep 26.

9.

Mucopolysaccharidosis VII in a Cat Caused by 2 Adjacent Missense Mutations in the GUSB Gene.

Wang P, Sorenson J, Strickland S, Mingus C, Haskins ME, Giger U.

J Vet Intern Med. 2015 Jul-Aug;29(4):1022-8. doi: 10.1111/jvim.13569. Epub 2015 Jun 25.

10.

Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.

Hinderer C, Bell P, Louboutin JP, Zhu Y, Yu H, Lin G, Choa R, Gurda BL, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Tarantal AF, Casal ML, Haskins ME, Wilson JM.

Mol Ther. 2015 Aug;23(8):1298-1307. doi: 10.1038/mt.2015.99. Epub 2015 May 29.

11.

A review of gene therapy in canine and feline models of lysosomal storage disorders.

Bradbury AM, Gurda BL, Casal ML, Ponder KP, Vite CH, Haskins ME.

Hum Gene Ther Clin Dev. 2015 Mar;26(1):27-37. doi: 10.1089/humc.2015.002. Epub 2015 Feb 11. Review.

12.

A review of gene therapy in canine and feline models of lysosomal storage disorders.

Bradbury AM, Gurda BL, Casal ML, Ponder K, Vite C, Haskins ME.

Hum Gene Ther Clin Dev. 2015 Jan 14. [Epub ahead of print]

PMID:
25587775
13.

Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I.

Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM.

Proc Natl Acad Sci U S A. 2014 Oct 14;111(41):14894-9. doi: 10.1073/pnas.1413645111. Epub 2014 Sep 29.

14.

Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I.

Hinderer C, Bell P, Gurda BL, Wang Q, Louboutin JP, Zhu Y, Bagel J, O'Donnell P, Sikora T, Ruane T, Wang P, Haskins ME, Wilson JM.

Mol Ther. 2014 Dec;22(12):2018-27. doi: 10.1038/mt.2014.135. Epub 2014 Jul 16.

15.

Effects of neonatal enzyme replacement therapy and simvastatin treatment on cervical spine disease in mucopolysaccharidosis I dogs.

Chiaro JA, O'Donnell P, Shore EM, Malhotra NR, Ponder KP, Haskins ME, Smith LJ.

J Bone Miner Res. 2014 Dec;29(12):2610-7. doi: 10.1002/jbmr.2290.

16.

Corrective GUSB transfer to the canine mucopolysaccharidosis VII cornea using a helper-dependent canine adenovirus vector.

Serratrice N, Cubizolle A, Ibanes S, Mestre-Francés N, Bayo-Puxan N, Creyssels S, Gennetier A, Bernex F, Verdier JM, Haskins ME, Couderc G, Malecaze F, Kalatzis V, Kremer EJ.

J Control Release. 2014 May 10;181:22-31. doi: 10.1016/j.jconrel.2014.02.022. Epub 2014 Mar 4.

17.

Corrective GUSB transfer to the canine mucopolysaccharidosis VII brain.

Cubizolle A, Serratrice N, Skander N, Colle MA, Ibanes S, Gennetier A, Bayo-Puxan N, Mazouni K, Mennechet F, Joussemet B, Cherel Y, Lajat Y, Vite C, Bernex F, Kalatzis V, Haskins ME, Kremer EJ.

Mol Ther. 2014 Apr;22(4):762-73. doi: 10.1038/mt.2013.283. Epub 2013 Dec 17.

18.

Diagnosis of feline acute intermittent porphyria presenting with erythrodontia requires molecular analyses.

Clavero S, Ahuja Y, Bishop DF, Kwait B, Haskins ME, Giger U, Desnick RJ.

Vet J. 2013 Dec;198(3):720-2. doi: 10.1016/j.tvjl.2013.10.008. Epub 2013 Oct 10.

19.

Adeno-associated virus serotypes 9 and rh10 mediate strong neuronal transduction of the dog brain.

Swain GP, Prociuk M, Bagel JH, O'Donnell P, Berger K, Drobatz K, Gurda BL, Haskins ME, Sands MS, Vite CH.

Gene Ther. 2014 Jan;21(1):28-36. doi: 10.1038/gt.2013.54. Epub 2013 Oct 17.

20.

The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade.

Bigg PW, Sleeper MM, O'Donnell PA, Liu Y, Wu S, Casal ML, Haskins ME, Ponder KP.

Mol Genet Metab. 2013 Nov;110(3):311-8. doi: 10.1016/j.ymgme.2013.06.015. Epub 2013 Jun 25.

21.

Pathogenesis of mitral valve disease in mucopolysaccharidosis VII dogs.

Bigg PW, Baldo G, Sleeper MM, O'Donnell PA, Bai H, Rokkam VR, Liu Y, Wu S, Giugliani R, Casal ML, Haskins ME, Ponder KP.

Mol Genet Metab. 2013 Nov;110(3):319-28. doi: 10.1016/j.ymgme.2013.06.013. Epub 2013 Jun 25.

22.

Acid ceramidase maintains the chondrogenic phenotype of expanded primary chondrocytes and improves the chondrogenic differentiation of bone marrow-derived mesenchymal stem cells.

Simonaro CM, Sachot S, Ge Y, He X, Deangelis VA, Eliyahu E, Leong DJ, Sun HB, Mason JB, Haskins ME, Richardson DW, Schuchman EH.

PLoS One. 2013 Apr 26;8(4):e62715. doi: 10.1371/journal.pone.0062715. Print 2013.

23.

The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade.

Xing EM, Knox VW, O'Donnell PA, Sikura T, Liu Y, Wu S, Casal ML, Haskins ME, Ponder KP.

Mol Genet Metab. 2013 Jun;109(2):183-93. doi: 10.1016/j.ymgme.2013.03.013. Epub 2013 Apr 6.

24.

Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I.

Vite CH, Nestrasil I, Mlikotic A, Jens JK, Snella EM, Gross W, Shapiro EG, Kovac V, Provenzale JM, Chen S, Le SQ, Kan SH, Banakar S, Wang RY, Haskins ME, Ellinwood NM, Dickson PI.

Comp Med. 2013 Apr;63(2):163-73.

25.

Postnatal progression of bone disease in the cervical spines of mucopolysaccharidosis I dogs.

Chiaro JA, Baron MD, Del Alcazar CM, O'Donnell P, Shore EM, Elliott DM, Ponder KP, Haskins ME, Smith LJ.

Bone. 2013 Jul;55(1):78-83. doi: 10.1016/j.bone.2013.03.014. Epub 2013 Apr 4.

26.

Dried blood spots for the enzymatic diagnosis of lysosomal storage diseases in dogs and cats.

Sewell AC, Haskins ME, Giger U.

Vet Clin Pathol. 2012 Dec;41(4):548-57. doi: 10.1111/j.1939-165x.2012.00485.x. Epub 2012 Nov 2.

27.

Pathogenesis of lumbar spine disease in mucopolysaccharidosis VII.

Smith LJ, Baldo G, Wu S, Liu Y, Whyte MP, Giugliani R, Elliott DM, Haskins ME, Ponder KP.

Mol Genet Metab. 2012 Sep;107(1-2):153-60. doi: 10.1016/j.ymgme.2012.03.014. Epub 2012 Mar 30.

28.

Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs.

Smith LJ, Martin JT, O'Donnell P, Wang P, Elliott DM, Haskins ME, Ponder KP.

Mol Genet Metab. 2012 Sep;107(1-2):145-52. doi: 10.1016/j.ymgme.2012.03.013. Epub 2012 Mar 29.

29.

Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.

Ponder KP, O'Malley TM, Wang P, O'Donnell PA, Traas AM, Knox VW, Aguirre GA, Ellinwood NM, Metcalf JA, Wang B, Parkinson-Lawrence EJ, Sleeper MM, Brooks DA, Hopwood JJ, Haskins ME.

Mol Ther. 2012 May;20(5):898-907. doi: 10.1038/mt.2012.9. Epub 2012 Mar 6.

30.

Renal failure associated with mucopolysaccharidosis type I in a cat from a MPS I research colony.

Cianciolo RE, Rhodes JL, Haskins ME, Clubb FJ, Lees GE.

Comp Med. 2011 Oct;61(5):441-4.

31.

Excessive activity of cathepsin K is associated with cartilage defects in a zebrafish model of mucolipidosis II.

Petrey AC, Flanagan-Steet H, Johnson S, Fan X, De la Rosa M, Haskins ME, Nairn AV, Moremen KW, Steet R.

Dis Model Mech. 2012 Mar;5(2):177-90. doi: 10.1242/dmm.008219. Epub 2011 Nov 1.

32.

Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates.

Bell P, Wang L, Gao G, Haskins ME, Tarantal AF, McCarter RJ, Zhu Y, Yu H, Wilson JM.

Mol Genet Metab. 2011 Nov;104(3):395-403. doi: 10.1016/j.ymgme.2011.06.002. Epub 2011 Jun 12.

33.

Animal models of lysosomal storage diseases: their development and clinical relevance.

Haskins ME, Giger U, Patterson DF.

In: Mehta A, Beck M, Sunder-Plassmann G, editors. Fabry Disease: Perspectives from 5 Years of FOS. Oxford: Oxford PharmaGenesis; 2006. Chapter 6.

34.

Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs.

Bell P, Gao G, Haskins ME, Wang L, Sleeper M, Wang H, Calcedo R, Vandenberghe LH, Chen SJ, Weisse C, Withnall E, Wilson JM.

Hum Gene Ther. 2011 Aug;22(8):985-97. doi: 10.1089/hum.2010.194. Epub 2011 Apr 11.

35.

Selective extraction and effective separation of galactosylsphingosine (psychosine) and glucosylsphingosine from other glycosphingolipids in pathological tissue samples.

Li YT, Li SC, Buck WR, Haskins ME, Wu SW, Khoo KH, Sidransky E, Bunnell BA.

Neurochem Res. 2011 Sep;36(9):1612-22. doi: 10.1007/s11064-010-0348-3. Epub 2010 Dec 7.

36.

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I.

Dickson PI, Hanson S, McEntee MF, Vite CH, Vogler CA, Mlikotic A, Chen AH, Ponder KP, Haskins ME, Tippin BL, Le SQ, Passage MB, Guerra C, Dierenfeld A, Jens J, Snella E, Kan SH, Ellinwood NM.

Mol Genet Metab. 2010 Oct-Nov;101(2-3):115-22. doi: 10.1016/j.ymgme.2010.06.020. Epub 2010 Jul 23.

37.

Feline congenital erythropoietic porphyria: two homozygous UROS missense mutations cause the enzyme deficiency and porphyrin accumulation.

Clavero S, Bishop DF, Giger U, Haskins ME, Desnick RJ.

Mol Med. 2010 Sep-Oct;16(9-10):381-8. doi: 10.2119/molmed.2010.00038. Epub 2010 May 12.

38.

Feline acute intermittent porphyria: a phenocopy masquerading as an erythropoietic porphyria due to dominant and recessive hydroxymethylbilane synthase mutations.

Clavero S, Bishop DF, Haskins ME, Giger U, Kauppinen R, Desnick RJ.

Hum Mol Genet. 2010 Feb 15;19(4):584-96. doi: 10.1093/hmg/ddp525. Epub 2009 Nov 24.

39.

Altered lumbar spine structure, biochemistry, and biomechanical properties in a canine model of mucopolysaccharidosis type VII.

Smith LJ, Martin JT, Szczesny SE, Ponder KP, Haskins ME, Elliott DM.

J Orthop Res. 2010 May;28(5):616-22. doi: 10.1002/jor.21030.

40.

In utero transplantation of monocytic cells in cats with alpha-mannosidosis.

Abkowitz JL, Sabo KM, Yang Z, Vite CH, Shields LE, Haskins ME.

Transplantation. 2009 Aug 15;88(3):323-9. doi: 10.1097/TP.0b013e3181b0d264.

41.

Altered olfactory epithelial structure and function in feline models of mucopolysaccharidoses I and VI.

Lischka FW, Gomez G, Yee KK, Dankulich-Nagrudny L, Lo L, Haskins ME, Rawson NE.

J Comp Neurol. 2008 Nov 20;511(3):360-72. doi: 10.1002/cne.21847.

42.

A single base pair mutation encoding a premature stop codon in the MIS type II receptor is responsible for canine persistent Müllerian duct syndrome.

Wu X, Wan S, Pujar S, Haskins ME, Schlafer DH, Lee MM, Meyers-Wallen VN.

J Androl. 2009 Jan-Feb;30(1):46-56. doi: 10.2164/jandrol.108.005736. Epub 2008 Aug 21.

43.

Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy.

Herati RS, Knox VW, O'Donnell P, D'Angelo M, Haskins ME, Ponder KP.

Mol Genet Metab. 2008 Nov;95(3):142-51. doi: 10.1016/j.ymgme.2008.07.003. Epub 2008 Aug 15.

44.

Clinical, electrophysiological, and serum biochemical measures of progressive neurological and hepatic dysfunction in feline Niemann-Pick type C disease.

Vite CH, Ding W, Bryan C, O'Donnell P, Cullen K, Aleman D, Haskins ME, Van Winkle T.

Pediatr Res. 2008 Nov;64(5):544-9. doi: 10.1203/PDR.0b013e318184d2ce.

45.

Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle.

Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, Le Guiner C.

Mol Ther. 2008 Jul;16(7):1291-1299. doi: 10.1038/mt.2008.87. Epub 2016 Dec 8.

PMID:
28178483
46.

Clinical characterization of cardiovascular abnormalities associated with feline mucopolysaccharidosis I and VI.

Sleeper MM, Kusiak CM, Shofer FS, O'Donnell P, Bryan C, Ponder KP, Haskins ME.

J Inherit Metab Dis. 2008 Jun;31(3):424-31. doi: 10.1007/s10545-008-0821-1. Epub 2008 May 23.

47.

CNS-directed gene therapy for lysosomal storage diseases.

Sands MS, Haskins ME.

Acta Paediatr. 2008 Apr;97(457):22-7. doi: 10.1111/j.1651-2227.2008.00660.x. Review.

48.

Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle.

Toromanoff A, Chérel Y, Guilbaud M, Penaud-Budloo M, Snyder RO, Haskins ME, Deschamps JY, Guigand L, Podevin G, Arruda VR, High KA, Stedman HH, Rolling F, Anegon I, Moullier P, Le Guiner C.

Mol Ther. 2008 Jul;16(7):1291-9. doi: 10.1038/mt.2008.87. Epub 2008 May 6.

49.

Gene therapy for cardiovascular manifestations of lysosomal storage diseases.

Sleeper MM, Haskins ME, Ponder KP.

Heart Metab. 2008;41:21-24.

50.

Mechanism of glycosaminoglycan-mediated bone and joint disease: implications for the mucopolysaccharidoses and other connective tissue diseases.

Simonaro CM, D'Angelo M, He X, Eliyahu E, Shtraizent N, Haskins ME, Schuchman EH.

Am J Pathol. 2008 Jan;172(1):112-22. Epub 2007 Dec 13.

Supplemental Content

Loading ...
Support Center