Wolfe Laboratory

Welcome to the Wolfe Lab, supervised by John H. Wolfe, Professor of Pathology and Medical Genetics in Pediatrics and Director, Walter Flato Goodman Center for Comparative Medical Genetics.

 Description of Research 

Our lab works on transferring disease correcting genes into the central nervous system (CNS) in animal models of human genetic diseases. In these diseases, the CNS is often not rescued by the therapies that help the rest of the body. The lab studies both ex vivo gene transfer into neural stem cells that are then transplanted and in vivo transfer using vectors injected directly into the brain.

Our studies involve comparisons of both the vectors used to introduce the genes into cells and the properties of the genes themselves. Additionally, we examine the ability of different cell types and subregions of the brain to be corrected. We also pursue new methods to follow the corrected cells and the expression of the correcting gene in the live animal using MRI and PET techniques. For a complete understanding of the therapy, we also are working on achieving a better understanding of the mechanism of these diseases in the brain.

Projects

Projects involve the molecular design and engineering of vectors, the understanding of the fate of vector-transferred genes in the brain, the regulation of gene expression from vectors, the biology of neural stem cell, the study of induced pluripotent stem cells (iPS), the use of imaging studies in genetic disease and gene therapy, and the proteomic and genomics analysis of the neurodegenerative brain. For students, projects can be tailored to interests, learning goals, and experience.

Contact Information

John H. Wolfe, PhD

Wolfe Lab Team
John Wolfe, PhD Dr. John H. Wolfe

Professor, Pathology and Medical Genetics, School of Veterinary Medicine and Department of Pediatrics, School of Medicine

Director, Walter Flato Goodman Center for Comparative Medical Genetics

Michael Parente, Wolfe Lab Michael Parente

Lab Manager

Faez Siddiqi, Wolfe Lab Faez Siddiqi, PhD

Research Associate

Contact:  siddiqif@email.chop.edu

Dr. Siddiqi's research interests are focused on understanding the molecular underpinnings of inherited neurodegenerative diseases, stem cell biology and CNS transplantation therapy. He has employed neurodevelopmental models to better appreciate endogenous stem cell diversity and subsequent lineage production that may become dysfunctional in the developed brain. His translational research goals concentrate on modifying immortalized and iPSC-derived neural stem cells with genetic factors to facilitate engraftment into the diseased brain. He utilizes a combination of cell culture techniques, genetic fate mapping, bioinformatics analysis, live-cell confocal imaging and transgenic animal studies with in vivo transplantation in these studies.

 Sea Young Yoon sm  Sea Young Yoon, PhD

 Research Associate

Contact:  seayoon@vet.upenn.edu

Dr. Yoon's research interests involve developing and optimizing viral gene therapy treatments. Her postdoctoral research involved AAV vector development and evaluation of novel AAV serotypes with alternative routes of vector administration to improve and optimize bio-distribution in the brain of mice and cats, and testing therapeutic efficacy of AAV-mediated gene transfer in cats affected with lysosomal storage diseases.

mayela-gayosso-sm Lucia Mayela Gayosso Miranda, PhD

Post-doctoral Fellow

Contact:  gayossoL@email.chop.edu

 
Dr. Brittney Gurda, Wolfe Lab  Brittney Gurda, VMD, PhD

 Post-doctoral Fellow

Contact:  gurdab@email.chop.edu

Dr. Gurda received her PhD in biochemistry and molecular biology from the University of Florida (2010) and her VMD in 2017. Her research has mainly focused on the adeno-associated virus as a gene therapy vector. These studies ranged from basic to translational including the three-dimensional structure and protein-protein interactions of the AAV capsid and its potential as a treatment for rare lysosomal storage diseases, such as the mucopolysaccharidoses and Niemann-Pick type C1. She attended veterinary school to better understand the pathophysiology of disease and to be able to use and understand animal models of human genetic diseases to their fullest capacity. Her studies will now focus on the ability of AAV vectors to utilize neuronal networks and how to exploit these properties in order to treat diseases of the central and peripheral nervous system.

 

Jacqueline Hunter, PhD

Post-doctoral Fellow

Dr. Hunter is a postdoctoral fellow in the Wolfe Laboratory. She received her PhD in Pharmacology & Toxicology from the University of the Sciences in Philadelphia. Her research focuses on evaluating the therapeutic efficacy of viral vector-mediated gene delivery to the brain using feline models of lysosomal storage disorders.

 
Trena Clarke, Wolfe Lab Trena Clarke

Research Technician IV

Ms. Clarke has over fourteen years experience as an Animal Care Technician in the Wolfe Lab at The Children's Hospital of Philadelphia Research Institute.  She works closely with the lab coordinating trainings and ensuring compliance with animal research protocols. She assists post doctoral researchers, graduate students and other lab personnel with organizing and maintaining animal models for projects. This includes genotyping, colony maintenance, surgical assistance and other scientific support in the lab and Research Institute.

Ara Polesky, Wolfe Lab Ara Polesky
Research Technician
  Emily Romick

Administrative Coordinator

Contact:  emromick@vet.upenn.edu or 215 590-7030

Siddiqi F, Wolfe JH. Stem Cell Therapy for the Central Nervous System in Lysosomal Storage Diseases. Hum Gene Ther. 2016 Jul 13. PubMed PMID: 27420186; PubMed Central PMCID: PMC5035913.

Parente MK, Rozen R, Seeholzer SH, Wolfe JH. Integrated analysis of proteome and transcriptome changes in the mucopolysaccharidosis type VII mouse hippocampus. Mol Genet Metab. 2016 May;118(1):41-54. 

Yoon SY, Bagel JH, O'Donnell PA, Vite CH, Wolfe JH. Clinical Improvement of Alpha-mannosidosis Cat Following a Single Cisterna Magna Infusion of AAV1. Mol Ther. 2016 Feb;24(1):26-33. 

Castle MJ, Turunen HT, Vandenberghe LH, Wolfe JH. Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids. Methods Mol Biol. 2016;1382:133-49. 

Kumar M, Duda JT, Yoon SY, Bagel J, O'Donnell P, Vite C, Pickup S, Gee JC, Wolfe JH, Poptani H. Diffusion Tensor Imaging for Assessing Brain Gray and White Matter Abnormalities in a Feline Model of α-Mannosidosis. J Neuropathol Exp Neurol. 2016 Jan;75(1):35-43. 

Griffin TA, Anderson HC, Wolfe JH. Ex vivo gene therapy using patient iPSC-derived NSCs reverses pathology in the brain of a homologous mouse model. Stem Cell Reports. 2015 May 12;4(5):835-46.  Epub 2015 Apr 9. 

Liu W, Griffin G, Clarke T, Parente MK, Valentino RJ, Wolfe JH, Fraser NW. Bilateral single-site intracerebral injection of a nonpathogenic herpes simplex virus-1 vector decreases anxiogenic behavior in MPS VII mice. Mol Ther Methods Clin Dev. 2015 Jan 28;2:14059. 

Castle MJ, Gershenson ZT, Giles AR, Holzbaur ELF, Wolfe JH.  Adeno-associated virus serotypes 1, 8, and 9 share conserved mechanisms for anterograde and retrograde axonal transport. Hum. Gene Ther. 2014.  25:  705-20. PMCID:  PMC4137353

Yoon SY, Gay-Antaki C, Ponde DE, Poptani H, Vite CH, and Wolfe JH.  Quantitative, non-invasive, in vivo longitudinal monitoring of gene expression in the brain by co-AAV transduction with a PET reporter gene.  Mol. Ther. Meth. & Clin. Devel. In Publication.

Castle MJ, Perlson E, Holzbaur ELF, and Wolfe JH.  Long-distance axonal transport of AAV9 is driven by dynein and kinesin-2 and is trafficked in a highly motile Rab7-positive compartment.  Mol. Ther. 2014. 22: 554-566. PMCID:  PMC3944332

Kumar M, Nasrallah IM, Kim S, Ittyerah R, Pickup S, Li J, Parente MK, Wolfe JH, and Poptani, H.  High-resolution magnetic resonance microscopy and diffusion tensor imaging to assess brain structural abnormalities in the murine mucopolysaccharidosis VII model.  J. Neuropath. Exp. Neurol. 2014. 73: 39-49. 

Weerakkody TN, Patel TP, Yue C, Takano H, Anderson HC, Meaney DF, Coulter DA, and Wolfe JH.  Engraftment of exogenous undifferentiated neural stem cells disrupts cortical network activity.  Mol. Ther. 2013. 21: 2258-2267. PMCID:  PMC3863790

Chaubey S, and Wolfe JH.  Transplantation of CD15-enriched murine neural stem cells leads to better survival and increased oligodendrocytes differentiation.  Stem Cells Transl. Med. 2013. 2: 444-454.  PMCID:  3574211 

Simonato M, Bennett J, Boulis NM, Castro MG, Fink DJ, Gray SJ, Lowenstein PR, Vandenberghe LH, Wilson TJ, Wolfe JH, and Glorioso, JC.  Gene therapy for neurological disorders.  Are we getting there?  Nat. Rev. Neurol. 2013. 9: 277-291 

Parente MK, Rozen R, Cearley CN, Wolfe JH. Dysregulation of gene expression in a lysosomal storage disease varies between brain regions implicating unexpected mechanisms of neuropathology. PLoS ONE. 2012;7(3):e32419.

Vandenberghe LH, Bell P, Maguire AM, Cearley CN, Xiao R, Calcedo R, Wang L, Castle MJ, Maguire AC, Grant R, Wolfe JH, Wilson JM, Bennett J. Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey. Sci Transl Med. 2011 Jun 22;3(88):88ra54. PubMed PMID: 21697530.

Magnitsky S, Vite CH, Delikatny EJ, Pickup S, Wehrli S, Wolfe JH, Poptani H. Magnetic resonance spectroscopy of the occipital cortex and the cerebellar vermis distinguishes individual cats affected with alpha-mannosidosis from normal cats.  NMR Biomed. 2010 Jan;23(1):74-9. PubMed PMID: 19743435; PubMed Central PMCID: PMC3045771.

Husain T, Passini MA, Parente MK, Fraser NW, Wolfe JH. Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is  similar to neural cell promoters. Gene Ther. 2009 Jul;16(7):927-32. Epub 2009 May 21. PubMed PMID: 19458648.

Walton RM, Magnitsky SG, Seiler GS, Poptani H, Wolfe JH. Transplantation and magnetic resonance imaging of canine neural progenitor cell grafts in the postnatal dog brain. J Neuropathol Exp Neurol. 2008 Oct;67(10):954-62. PubMed PMID: 18800012; PubMed Central PMCID: PMC2856607.

Magnitsky S, Walton RM, Wolfe JH, Poptani H. Magnetic resonance imaging detects differences in migration between primary and immortalized neural stem cells. Acad Radiol. 2008 Oct;15(10):1269-81. PubMed PMID: 18790399; PubMed Central PMCID: PMC2573997.

Cearley CN, Vandenberghe LH, Parente MK, Carnish ER, Wilson JM, Wolfe JH. Expanded repertoire of AAV vector serotypes mediate unique patterns of transduction in mouse brain. Mol Ther. 2008 Oct;16(10):1710-8. Epub 2008 Aug 19.  PubMed PMID: 18714307; PubMed Central PMCID: PMC3056207.

Walton RM, Wolfe JH. In vitro growth and differentiation of canine olfactory bulb-derived neural progenitor cells under variable culture conditions. J Neurosci Methods. 2008 Mar 30;169(1):158-67. Epub 2007 Dec 27. PubMed PMID: 18261803; PubMed Central PMCID: PMC2377027.

Vite CH, Magnitsky S, Aleman D, O'Donnell P, Cullen K, Ding W, Pickup S, Wolfe JH, Poptani H. Apparent diffusion coefficient reveals gray and white matter disease, and T2 mapping detects white matter disease in the brain in feline alpha-mannosidosis. AJNR Am J Neuroradiol. 2008 Feb;29(2):308-13. Epub 2007 Nov 1. PubMed PMID: 17974615.

Walton RM, Wolfe JH. Abnormalities in neural progenitor cells in a dog model of lysosomal storage disease. J Neuropathol Exp Neurol. 2007 Aug;66(8):760-9. PubMed PMID: 17882020.

Cearley CN, Wolfe JH. A single injection of an adeno-associated virus vector into nuclei with divergent connections results in widespread vector distribution in the brain and global correction of a neurogenetic disease. J Neurosci. 2007 Sep 12;27(37):9928-40. PubMed PMID: 17855607.

Magnitsky S, Walton RM, Wolfe JH, Poptani H. Magnetic resonance imaging as a  tool for monitoring stem cell migration. Neurodegener Dis. 2007;4(4):314-21. Review. PubMed PMID: 17627135.

Berges BK, Wolfe JH, Fraser NW. Transduction of brain by herpes simplex virus vectors. Mol Ther. 2007 Jan;15(1):20-9. Review. PubMed PMID: 17164771.

Watson DJ, Walton RM, Magnitsky SG, Bulte JW, Poptani H, Wolfe JH. Structure-specific patterns of neural stem cell engraftment after transplantation in the adult mouse brain. Hum Gene Ther. 2006 Jul;17(7):693-704. PubMed PMID: 16839269.

Karolewski BA, Wolfe JH. Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII. Mol Ther. 2006 Jul;14(1):14-24. Epub 2006 Apr 19. PubMed PMID: 16624622.

Li LB, Toan SV, Zelenaia O, Watson DJ, Wolfe JH, Rothstein JD, Robinson MB. Regulation of astrocytic glutamate transporter expression by Akt: evidence for a selective transcriptional effect on the GLT-1/EAAT2 subtype. J Neurochem. 2006 May;97(3):759-71. Epub 2006 Mar 29. PubMed PMID: 16573655.

Berges BK, Yellayi S, Karolewski BA, Miselis RR, Wolfe JH, Fraser NW. Widespread correction of lysosomal storage in the mucopolysaccharidosis type VII mouse brain with a herpes simplex virus type 1 vector expressing beta-glucuronidase. Mol Ther. 2006 May;13(5):859-69. Epub 2006 Mar 3. PubMed PMID: 16515890.

Cearley CN, Wolfe JH. Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain. Mol Ther. 2006 Mar;13(3):528-37. Epub 2006 Jan 18. PubMed PMID: 16413228.

Berges BK, Wolfe JH, Fraser NW. Stable levels of long-term transgene expression driven by the latency-associated transcript promoter in a herpes simplex virus type 1 vector. Mol Ther. 2005 Dec;12(6):1111-9. Epub 2005 Aug 24. PubMed PMID: 16122987.

Magnitsky S, Watson DJ, Walton RM, Pickup S, Bulte JW, Wolfe JH, Poptani H. In vivo and ex vivo MRI detection of localized and disseminated neural stem cell grafts in the mouse brain. Neuroimage. 2005 Jul 1;26(3):744-54. Epub 2005 Apr 7. PubMed PMID: 15955483.

Jiang K, Watson DJ, Wolfe JH. A genetic fusion construct between the tetanus toxin C fragment and the lysosomal acid hydrolase beta-glucuronidase expresses a bifunctional protein with enhanced secretion and neuronal uptake. J Neurochem. 2005 Jun;93(5):1334-44. PubMed PMID: 15934952.

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. Ann Neurol. 2005 Mar;57(3):355-64. PubMed PMID: 15732095.

Watson DJ, Passini MA, Wolfe JH. Transduction of the choroid plexus and ependyma in neonatal mouse brain by vesicular stomatitis virus glycoprotein-pseudotyped lentivirus and adeno-associated virus type 5 vectors. Hum Gene Ther. 2005 Jan;16(1):49-56. PubMed PMID: 15703488.

Susarla BT, Seal RP, Zelenaia O, Watson DJ, Wolfe JH, Amara SG, Robinson MB. Differential regulation of GLAST immunoreactivity and activity by protein kinase C: evidence for modification of amino and carboxyl termini. J Neurochem. 2004 Dec;91(5):1151-63. PubMed PMID: 15569258.

Watson DJ, Karolewski BA, Wolfe JH. Stable gene delivery to CNS cells using lentiviral vectors. Methods Mol Biol. 2004;246:413-28. PubMed PMID: 14970607.

Passini MA, Watson DJ, Wolfe JH. Gene delivery to the mouse brain with adeno-associated virus. Methods Mol Biol. 2004;246:225-36. PubMed PMID: 14970596.

Karolewski BA, Watson DJ, Parente MK, Wolfe JH. Comparison of transfection conditions for a lentivirus vector produced in large volumes. Hum Gene Ther. 2003 Sep 20;14(14):1287-96. PubMed PMID: 14503964.

Vite CH, Passini MA, Haskins ME, Wolfe JH. Adeno-associated virus vector-mediated transduction in the cat brain. Gene Ther. 2003 Oct;10(22):1874-81. PubMed PMID: 14502216.

Passini MA, Watson DJ, Vite CH, Landsburg DJ, Feigenbaum AL, Wolfe JH. Intraventricular brain injection of adeno-associated virus type 1 (AAV1) in neonatal mice results in complementary patterns of neuronal transduction to AAV2 and total long-term correction of storage lesions in the brains of beta-glucuronidase-deficient mice. J Virol. 2003 Jun;77(12):7034-40. PubMed PMID: 12768022; PubMed Central PMCID: PMC156185.

Watson DJ, Longhi L, Lee EB, Fulp CT, Fujimoto S, Royo NC, Passini MA, Trojanowski JQ, Lee VM, McIntosh TK, Wolfe JH. Genetically modified NT2N human neuronal cells mediate long-term gene expression as CNS grafts in vivo and improve functional cognitive outcome following experimental traumatic brain injury. J Neuropathol Exp Neurol. 2003 Apr;62(4):368-80. PubMed PMID: 12722829.

Watson DJ, Wolfe JH. Lentiviral vectors for gene transfer to the central nervous system. Applications in lysosomal storage disease animal models. Methods Mol Med. 2003;76:383-403. PubMed PMID: 12526176.

Heuer GG, Passini MA, Jiang K, Parente MK, Lee VM, Trojanowski JQ, Wolfe JH. Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible. Ann Neurol. 2002 Dec;52(6):762-70. PubMed PMID: 12447930.

Prasad Alur RK, Foley B, Parente MK, Tobin DK, Heuer GG, Avadhani AN, Pongubala J, Wolfe JH. Modification of multiple transcriptional regulatory elements in a Moloney murine leukemia virus gene transfer vector circumvents silencing in fibroblast grafts and increases levels of expression of the transferred enzyme. Gene Ther. 2002 Sep;9(17):1146-54. Erratum in: Gene Ther 2002 Dec;9(23):1645. PubMed PMID: 12170378.

Passini MA, Lee EB, Heuer GG, Wolfe JH. Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream. J Neurosci. 2002 Aug 1;22(15):6437-46. PubMed PMID: 12151523.

Nagano M, Watson DJ, Ryu BY, Wolfe JH, Brinster RL. Lentiviral vector transduction of male germ line stem cells in mice. FEBS Lett. 2002 Jul 31;524(1-3):111-5. PubMed PMID: 12135751.

Watson DJ, Kobinger GP, Passini MA, Wilson JM, Wolfe JH. Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins. Mol Ther. 2002 May;5(5 Pt 1):528-37. PubMed PMID: 11991743.

Passini MA, Wolfe JH. Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector. J Virol. 2001 Dec;75(24):12382-92. PubMed PMID: 11711628; PubMed Central PMCID: PMC116134.

Springer SL, Vite CH, Polesky AC, Kesari S, Fraser NW, Wolfe JH. Infection and establishment of latency in the dog brain after direct inoculation of a nonpathogenic strain of herpes simplex virus-1. J Neurovirol. 2001 Apr;7(2):149-54. PubMed PMID: 11517387.

Vite CH, McGowan JC, Braund KG, Drobatz KJ, Glickson JD, Wolfe JH, Haskins ME. Histopathology, electrodiagnostic testing, and magnetic resonance imaging show significant peripheral and central nervous system myelin abnormalities in the cat model of alpha-mannosidosis. J Neuropathol Exp Neurol. 2001 Aug;60(8):817-28. PubMed PMID: 11487056.

Casal ML, Wolfe JH. In utero transplantation of fetal liver cells in the mucopolysaccharidosis type VII mouse results in low-level chimerism, but overexpression of beta-glucuronidase can delay onset of clinical signs. Blood. 2001 Mar 15;97(6):1625-34. PubMed PMID: 11238101.

Sun H, Wolfe JH. Characterization of an unusual variant mRNA of human lysosomal alpha-mannosidase. Exp Mol Med. 2000 Dec 31;32(4):187-92. PubMed PMID: 11190268.

Wolfe JH, Sands MS, Harel N, Weil MA, Parente MK, Polesky AC, Reilly JJ, Hasson C, Weimelt S, Haskins ME. Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of  mucopolysaccharidosis VII. Mol Ther. 2000 Dec;2(6):552-61. PubMed PMID: 11124056.

Zhu J, Kang W, Wolfe JH, Fraser NW. Significantly increased expression of beta-glucuronidase in the central nervous system of mucopolysaccharidosis type VII mice from the latency-associated transcript promoter in a nonpathogenic herpes simplex virus type 1 vector. Mol Ther. 2000 Jul;2(1):82-94. PubMed PMID: 10899831.

Casal ML, Wolfe JH. Mucopolysaccharidosis type VII in the developing mouse fetus. Pediatr Res. 2000 Jun;47(6):750-6. PubMed PMID: 10832732.

Skorupa AF, Fisher KJ, Wilson JM, Parente MK, Wolfe JH. Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice. Exp Neurol. 1999 Nov;160(1):17-27. PubMed PMID: 10630187.

Ginsberg SD, Galvin JE, Lee VM, Rorke LB, Dickson DW, Wolfe JH, Jones MZ, Trojanowski JQ. Accumulation of intracellular amyloid-beta peptide (A beta 1-40) in mucopolysaccharidosis brains. J Neuropathol Exp Neurol. 1999 Aug;58(8):815-24. PubMed PMID: 10446806.

Sun H, Yang M, Haskins ME, Patterson DF, Wolfe JH. Retrovirus vector-mediated correction and cross-correction of lysosomal alpha-mannosidase deficiency in human and feline fibroblasts. Hum Gene Ther. 1999 May 20;10(8):1311-9. PubMed PMID: 10365662.

Casal ML, Wolfe JH. Variant clinical course of mucopolysaccharidosis type VII in two groups of mice carrying the same mutation. Lab Invest. 1998 Dec;78(12):1575-81. PubMed PMID: 9881957.

Taylor RM, Wolfe JH. Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase. Nat Med. 1997 Jul;3(7):771-4. PubMed PMID: 9212105.

Taylor RM, Wolfe JH. Glycosaminoglycan storage in cultured neonatal murine mucopolysaccharidosis type VII neuroglial cells and correction by beta-glucuronidase gene transfer. J Neurochem. 1997 May;68(5):2079-85. PubMed PMID: 9109535.

Wolfe JH, Martin CE, Deshmane SL, Reilly JJ, Kesari S, Fraser NW. Increased susceptibility to the pathogenic effects of wild-type and recombinant herpesviruses in MPS VII mice compared to normal siblings. J Neurovirol. 1996 Dec;2(6):417-22. PubMed PMID: 8972424.

Parente MK, Wolfe JH. Production of increased titer retrovirus vectors from stable producer cell lines by superinfection and concentration. Gene Ther. 1996 Sep;3(9):756-60. PubMed PMID: 8875222.

Smith BF, Stedman H, Rajpurohit Y, Henthorn PS, Wolfe JH, Patterson DF, Giger U. Molecular basis of canine muscle type phosphofructokinase deficiency. J Biol Chem. 1996 Aug 16;271(33):20070-4. PubMed PMID: 8702726.

Smith BF, Henthorn PS, Rajpurohit Y, Stedman H, Wolfe JH, Patterson DF, Giger U. A cDNA encoding canine muscle-type phosphofructokinase. Gene. 1996 Feb 12;168(2):275-6. PubMed PMID: 8654960.

Wolfe JH, Kyle JW, Sands MS, Sly WS, Markowitz DG, Parente MK. High level expression and export of beta-glucuronidase from murine mucopolysaccharidosis VII cells corrected by a double-copy retrovirus vector. Gene Ther. 1995 Jan;2(1):70-8. PubMed PMID: 7712336.

Taylor RM, Wolfe JH. Cross-correction of beta-glucuronidase deficiency by retroviral vector-mediated gene transfer. Exp Cell Res. 1994 Oct;214(2):606-13. PubMed PMID: 7925654.

Wolfe JH. Recent progress in gene therapy for inherited diseases. Curr Opin Pediatr. 1994 Apr;6(2):213-8. Review. PubMed PMID: 8032403.

Wolfe JH, Taylor RM, Sands MS, Haskins ME, Patterson DF, Snyder EY. Mucopolysaccharidosis type VII as a model system for gene transfer to the central nervous system. Gene Ther. 1994;1 Suppl 1:S55. PubMed PMID: 8542400.

Wolfe JH, Deshmane SL, Fraser NW. Herpesvirus vector gene transfer and expression of beta-glucuronidase in the central nervous system of MPS VII mice. Nat Genet. 1992 Aug;1(5):379-84. PubMed PMID: 1338772.

Smith BF, Hoffman RK, Giger U, Wolfe JH. Genes transferred by retroviral vectors into normal and mutant myoblasts in primary cultures are expressed in myotubes. Mol Cell Biol. 1990 Jun;10(6):3268-71. PubMed PMID: 2160598; PubMed Central PMCID: PMC360696.

Stramm LE, Wolfe JH, Schuchman EH, Haskins ME, Patterson DF, Aguirre GD. Beta-glucuronidase mediated pathway essential for retinal pigment epithelial degradation of glycosaminoglycans. Disease expression and in vitro disease correction using retroviral mediated cDNA transfer. Exp Eye Res. 1990 May;50(5):521-32. PubMed PMID: 2164946.

Wolfe JH, Schuchman EH, Stramm LE, Concaugh EA, Haskins ME, Aguirre GD, Patterson DF, Desnick RJ, Gilboa E. Restoration of normal lysosomal function in mucopolysaccharidosis type VII cells by retroviral vector-mediated gene transfer. Proc Natl Acad Sci U S A. 1990 Apr;87(8):2877-81. PubMed PMID: 2158095; PubMed Central PMCID: PMC53796.

Patterson DF, Haskins ME, Jezyk PF, Giger U, Meyers-Wallen VN, Aguirre G, Fyfe JC, Wolfe JH. Research on genetic diseases: reciprocal benefits to animals and man. J Am Vet Med Assoc. 1988 Nov 1;193(9):1131-44. Review. PubMed PMID: 3058665.

Wolfe JH, Halliwell RE. Total hemolytic complement values in normal and diseased dog populations. Vet Immunol Immunopathol. 1980 Aug;1(3):287-98. PubMed  PMID: 15615062.