| Year |
Citation |
Score |
| 2017 |
Hinderer C, Bell P, Katz N, Vite C, Louboutin JP, Bote E, Yu H, Zhu Y, Casal ML, Bagel J, O'Donnell P, Wang P, Haskins ME, Goode T, Wilson JM. Evaluation of intrathecal routes of administration for adeno-associated virus vectors in large animals. Human Gene Therapy. PMID 28806897 DOI: 10.1089/Hum.2017.026 |
0.337 |
|
| 2016 |
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. Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model. Molecular Genetics and Metabolism. PMID 27386755 DOI: 10.1016/J.Ymgme.2016.06.006 |
0.363 |
|
| 2016 |
Callan MB, Haskins ME, Wang P, Zhou S, High KA, Arruda VR. Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs. Plos One. 11: e0151800. PMID 27011017 DOI: 10.1371/Journal.Pone.0151800 |
0.387 |
|
| 2015 |
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, ... ... Haskins ME, et al. Evaluation of AAV-mediated gene therapy for central nervous system disease in canine mucopolysaccharidosis VII. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 26447927 DOI: 10.1038/Mt.2015.189 |
0.363 |
|
| 2015 |
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, et al. Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 26022732 DOI: 10.1038/Mt.2015.99 |
0.437 |
|
| 2015 |
Bradbury AM, Gurda BL, Casal ML, Ponder KP, Vite CH, Haskins ME. A review of gene therapy in canine and feline models of lysosomal storage disorders. Human Gene Therapy. Clinical Development. 26: 27-37. PMID 25671613 DOI: 10.1089/Humc.2015.002 |
0.382 |
|
| 2014 |
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. Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I. Proceedings of the National Academy of Sciences of the United States of America. 111: 14894-9. PMID 25267637 DOI: 10.1073/Pnas.1413645111 |
0.408 |
|
| 2014 |
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. Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 2018-27. PMID 25027660 DOI: 10.1038/Mt.2014.135 |
0.419 |
|
| 2013 |
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. Pathogenesis of mitral valve disease in mucopolysaccharidosis VII dogs. Molecular Genetics and Metabolism. 110: 319-28. PMID 23856419 DOI: 10.1016/j.ymgme.2013.06.013 |
0.314 |
|
| 2013 |
Xing EM, Knox VW, O'Donnell PA, Sikura T, Liu Y, Wu S, Casal ML, Haskins ME, Ponder KP. The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade. Molecular Genetics and Metabolism. 109: 183-93. PMID 23628461 DOI: 10.1016/j.ymgme.2013.03.013 |
0.461 |
|
| 2013 |
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, et al. Features of brain MRI in dogs with treated and untreated mucopolysaccharidosis type I. Comparative Medicine. 63: 163-73. PMID 23582423 |
0.647 |
|
| 2013 |
Ferla R, O'Malley T, Calcedo R, O'Donnell P, Wang P, Cotugno G, Claudiani P, Wilson JM, Haskins M, Auricchio A. Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8. Human Gene Therapy. 24: 163-9. PMID 23194248 DOI: 10.1089/Hum.2012.179 |
0.359 |
|
| 2012 |
Smith LJ, Martin JT, O'Donnell P, Wang P, Elliott DM, Haskins ME, Ponder KP. Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs. Molecular Genetics and Metabolism. 107: 145-52. PMID 22510705 DOI: 10.1016/J.Ymgme.2012.03.013 |
0.448 |
|
| 2012 |
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. Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 898-907. PMID 22395531 DOI: 10.1038/mt.2012.9 |
0.687 |
|
| 2011 |
Bell P, Wang L, Gao G, Haskins ME, Tarantal AF, McCarter RJ, Zhu Y, Yu H, Wilson JM. Inverse zonation of hepatocyte transduction with AAV vectors between mice and non-human primates. Molecular Genetics and Metabolism. 104: 395-403. PMID 21778099 DOI: 10.1016/J.Ymgme.2011.06.002 |
0.348 |
|
| 2011 |
Sleeper M, Bish LT, Haskins M, Ponder KP, Sweeney HL. Status of therapeutic gene transfer to treat cardiovascular disease in dogs and cats. Journal of Veterinary Cardiology : the Official Journal of the European Society of Veterinary Cardiology. 13: 131-40. PMID 21640678 DOI: 10.1016/j.jvc.2011.04.001 |
0.305 |
|
| 2011 |
Vite CH, Wang P, Patel RT, Walton RM, Walkley SU, Sellers RS, Ellinwood NM, Cheng AS, White JT, O'Neill CA, Haskins M. Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations. Molecular Genetics and Metabolism. 103: 268-74. PMID 21482164 DOI: 10.1016/J.Ymgme.2011.03.011 |
0.635 |
|
| 2011 |
Bell P, Gao G, Haskins ME, Wang L, Sleeper M, Wang H, Calcedo R, Vandenberghe LH, Chen SJ, Weisse C, Withnall E, Wilson JM. Evaluation of adeno-associated viral vectors for liver-directed gene transfer in dogs. Human Gene Therapy. 22: 985-97. PMID 21204705 DOI: 10.1089/Hum.2010.194 |
0.37 |
|
| 2011 |
Cotugno G, Annunziata P, Tessitore A, O'Malley T, Capalbo A, Faella A, Bartolomeo R, O'Donnell P, Wang P, Russo F, Sleeper MM, Knox VW, Fernandez S, Levanduski L, Hopwood J, ... ... Haskins M, et al. Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 461-9. PMID 21119624 DOI: 10.1038/Mt.2010.257 |
0.371 |
|
| 2010 |
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, et al. Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I. Molecular Genetics and Metabolism. 101: 115-22. PMID 20655780 DOI: 10.1016/j.ymgme.2010.06.020 |
0.612 |
|
| 2010 |
Metcalf JA, Linders B, Wu S, Bigg P, O'Donnell P, Sleeper MM, Whyte MP, Haskins M, Ponder KP. Upregulation of elastase activity in aorta in mucopolysaccharidosis I and VII dogs may be due to increased cytokine expression. Molecular Genetics and Metabolism. 99: 396-407. PMID 20044292 DOI: 10.1016/j.ymgme.2009.12.003 |
0.317 |
|
| 2009 |
Haskins M. Gene therapy for lysosomal storage diseases (LSDs) in large animal models. Ilar Journal / National Research Council, Institute of Laboratory Animal Resources. 50: 112-21. PMID 19293456 |
0.34 |
|
| 2008 |
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, et al. Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1291-1299. PMID 28178483 DOI: 10.1038/Mt.2008.87 |
0.545 |
|
| 2008 |
Herati RS, Knox VW, O'Donnell P, D'Angelo M, Haskins ME, Ponder KP. Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy. Molecular Genetics and Metabolism. 95: 142-51. PMID 18707908 DOI: 10.1016/j.ymgme.2008.07.003 |
0.43 |
|
| 2008 |
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, et al. Safety and efficacy of regional intravenous (r.i.) versus intramuscular (i.m.) delivery of rAAV1 and rAAV8 to nonhuman primate skeletal muscle. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1291-9. PMID 18461055 DOI: 10.1038/mt.2008.87 |
0.53 |
|
| 2008 |
Sands MS, Haskins ME. CNS-directed gene therapy for lysosomal storage diseases. Acta Paediatrica (Oslo, Norway : 1992). Supplement. 97: 22-7. PMID 18339183 DOI: 10.1111/j.1651-2227.2008.00660.x |
0.348 |
|
| 2008 |
Tessitore A, Faella A, O'Malley T, Cotugno G, Doria M, Kunieda T, Matarese G, Haskins M, Auricchio A. Biochemical, pathological, and skeletal improvement of mucopolysaccharidosis VI after gene transfer to liver but not to muscle. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 30-7. PMID 17955027 DOI: 10.1038/Sj.Mt.6300325 |
0.303 |
|
| 2007 |
Traas AM, Wang P, Ma X, Tittiger M, Schaller L, O'donnell P, Sleeper MM, Vite C, Herati R, Aguirre GD, Haskins M, Ponder KP. Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 1423-31. PMID 17519893 DOI: 10.1038/sj.mt.6300201 |
0.441 |
|
| 2007 |
Ellinwood NM, Colle MA, Weil MA, Casal ML, Vite CH, Wiemelt S, Hasson CW, O'Malley TM, He X, Prociuk U, Verot L, Melniczek JR, Lannon A, Aguirre GD, Knox VW, ... ... Haskins ME, et al. Bone marrow transplantation for feline mucopolysaccharidosis I. Molecular Genetics and Metabolism. 91: 239-50. PMID 17482862 DOI: 10.1016/J.Ymgme.2007.03.001 |
0.636 |
|
| 2007 |
Xu L, Mei M, Haskins ME, Nichols TC, O'donnell P, Cullen K, Dillow A, Bellinger D, Ponder KP. Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice. Thrombosis Research. 120: 269-80. PMID 17095052 DOI: 10.1016/j.thromres.2006.09.010 |
0.306 |
|
| 2006 |
Ponder KP, Wang B, Wang P, Ma X, Herati R, Wang B, Cullen K, O'Donnell P, Ellinwood NM, Traas A, Primeau TM, Haskins ME. Mucopolysaccharidosis I cats mount a cytotoxic T lymphocyte response after neonatal gene therapy that can be blocked with CTLA4-Ig. Molecular Therapy : the Journal of the American Society of Gene Therapy. 14: 5-13. PMID 16698321 DOI: 10.1016/j.ymthe.2006.03.015 |
0.669 |
|
| 2006 |
Casal M, Haskins M. Large animal models and gene therapy. European Journal of Human Genetics : Ejhg. 14: 266-72. PMID 16333317 DOI: 10.1038/sj.ejhg.5201535 |
0.309 |
|
| 2006 |
Wang B, O'Malley TM, Xu L, Vite C, Wang P, O'Donnell PA, Ellinwood NM, Haskins ME, Ponder KP. Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs. Molecular Genetics and Metabolism. 87: 8-21. PMID 16275036 DOI: 10.1016/j.ymgme.2005.08.014 |
0.682 |
|
| 2005 |
Vite CH, McGowan JC, Niogi SN, Passini MA, Drobatz KJ, Haskins ME, Wolfe JH. Effective gene therapy for an inherited CNS disease in a large animal model. Annals of Neurology. 57: 355-64. PMID 15732095 DOI: 10.1002/Ana.20392 |
0.333 |
|
| 2004 |
Xu L, O'Malley T, Sands MS, Wang B, Meyerrose T, Haskins ME, Ponder KP. In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 10: 37-44. PMID 15233940 DOI: 10.1016/J.Ymthe.2004.04.010 |
0.372 |
|
| 2004 |
Ellinwood NM, Vite CH, Haskins ME. Gene therapy for lysosomal storage diseases: the lessons and promise of animal models. The Journal of Gene Medicine. 6: 481-506. PMID 15133760 DOI: 10.1002/jgm.581 |
0.645 |
|
| 2004 |
Mango RL, Xu L, Sands MS, Vogler C, Seiler G, Schwarz T, Haskins ME, Ponder KP. Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs. Molecular Genetics and Metabolism. 82: 4-19. PMID 15110316 DOI: 10.1016/j.ymgme.2004.01.015 |
0.325 |
|
| 2004 |
Zhang J, Xu L, Haskins ME, Parker Ponder K. Neonatal gene transfer with a retroviral vector results in tolerance to human factor IX in mice and dogs. Blood. 103: 143-51. PMID 12969967 DOI: 10.1182/blood-2003-06-2181 |
0.308 |
|
| 2002 |
Xu L, Mango RL, Sands MS, Haskins ME, Ellinwood NM, Ponder KP. Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 6: 745-58. PMID 12498771 DOI: 10.1006/mthe.2002.0809 |
0.689 |
|
| 2002 |
Ponder KP, Melniczek JR, Xu L, Weil MA, O'Malley TM, O'Donnell PA, Knox VW, Aguirre GD, Mazrier H, Ellinwood NM, Sleeper M, Maguire AM, Volk SW, Mango RL, Zweigle J, ... ... Haskins ME, et al. Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs. Proceedings of the National Academy of Sciences of the United States of America. 99: 13102-7. PMID 12232044 DOI: 10.1073/pnas.192353499 |
0.699 |
|
| 2002 |
Licht T, Haskins M, Henthorn P, Kleiman SE, Bodine DM, Whitwam T, Puck JM, Gottesman MM, Melniczek JR. Drug selection with paclitaxel restores expression of linked IL-2 receptor gamma -chain and multidrug resistance (MDR1) transgenes in canine bone marrow. Proceedings of the National Academy of Sciences of the United States of America. 99: 3123-8. PMID 11867757 DOI: 10.1073/Pnas.052712199 |
0.338 |
|
| 2002 |
Xu L, Haskins ME, Melniczek JR, Gao C, Weil MA, O'Malley TM, O'Donnell PA, Mazrier H, Ellinwood NM, Zweigle J, Wolfe JH, Ponder KP. Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs. Molecular Therapy : the Journal of the American Society of Gene Therapy. 5: 141-53. PMID 11829521 DOI: 10.1006/mthe.2002.0527 |
0.671 |
|
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