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David Wenger

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Mohammad A Rafi; Han Zhi Rao; Mark T Curtis; Paola Luzi; David A Wenger
Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease.
Paola Luzi; Ronnie M Abraham; Mohammad A Rafi; Mark Curtis; D Craig Hooper; David A Wenger
Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy.
Michelina Strazza; Alice Luddi; Marica Carbone; Mohammad A Rafi; Elvira Costantino-Ceccarini; David A Wenger
Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells.

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All Publications

2012: Mohammad A Rafi; Han Zhi Rao; Mark T Curtis; Paola Luzi; David A Wenger
Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease.
Molecular therapy : the journal of the American Society of Gene Therapy 2012;20(11):2031-42.
2009: Paola Luzi; Ronnie M Abraham; Mohammad A Rafi; Mark Curtis; D Craig Hooper; David A Wenger
Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy.
Brain research 2009;1300():146-58.
2009: Michelina Strazza; Alice Luddi; Marica Carbone; Mohammad A Rafi; Elvira Costantino-Ceccarini; David A Wenger
Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells.
Molecular genetics and metabolism 2009;97(1):27-34.
2005: Mariam Zaka; Mohammad A Rafi; Han Zhi Rao; Paola Luzi; David A Wenger
Insulin-like growth factor-1 provides protection against psychosine-induced apoptosis in cultured mouse oligodendrocyte progenitor cells using primarily the PI3K/Akt pathway.
Molecular and cellular neurosciences 2005;30(3):398-407.
2005: Steven U Walkley; Mary Anna Thrall; Mark E Haskins; TW Mitchell; David A Wenger; DE Brown; S Dial; H Seim
Abnormal neuronal metabolism and storage in mucopolysaccharidosis type VI (Maroteaux-Lamy) disease.
Neuropathology and applied neurobiology 2005;31(5):536-44.
2005: Paola Luzi; Mohammad A Rafi; Mariam Zaka; Han Zhi Rao; Mark Curtis; marie T vanier; David A Wenger
Biochemical and pathological evaluation of long-lived mice with globoid cell leukodystrophy after bone marrow transplantation.
Molecular genetics and metabolism 2005;86(1-2):150-9.
2005: Darshong Lin; Corinne R Fantz; Beth Levy; Mohammad A Rafi; Carole Vogler; David A Wenger; Mark S Sands
AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2.
Molecular therapy : the journal of the American Society of Gene Therapy 2005;12(3):422-30.
2005: Mohammad A Rafi; Han Zhi Rao; Marco A Passini; Mark Curtis; marie T vanier; Mariam Zaka; Paola Luzi; John H Wolfe; David A Wenger
AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy.
Molecular therapy : the journal of the American Society of Gene Therapy 2005;11(5):734-44.
2004: Paola Luzi; Mariam Zaka; Han Zhi Rao; Mark Curtis; Mohammad A Rafi; David A Wenger
Generation of transgenic mice expressing insulin-like growth factor-1 under the control of the myelin basic protein promoter: increased myelination and potential for studies on the effects of increased IGF-1 on experimentally and genetically induced demyelination.
Neurochemical research 2004;29(5):881-9.
2004: Mariam Zaka; David A Wenger
Psychosine-induced apoptosis in a mouse oligodendrocyte progenitor cell line is mediated by caspase activation.
Neuroscience letters 2004;358(3):205-9.
2003: Zahida Parveen; Muhammad Mukhtar; Mohammad A Rafi; David A Wenger; Khwaja M Siddiqui; Catherine A Siler; Bernhard Dietzschold; Roger J Pomerantz; Matthias J Schnell; Ralph Dornburg
Cell-type-specific gene delivery into neuronal cells in vitro and in vivo.
Virology 2003;314(1):74-83.
2003: Kyra L Somers; Michael A Royals; Eugene D Carstea; Mohammad A Rafi; David A Wenger; Mary Anna Thrall
Mutation analysis of feline Niemann-Pick C1 disease.
Molecular genetics and metabolism 2003;79(2):99-103.
2003: Mohammad A Rafi; Stephanie Coppola; Shu Ling Liu; Han Zhi Rao; David A Wenger
Disease-causing mutations in cis with the common arylsulfatase A pseudodeficiency allele compound the difficulties in accurately identifying patients and carriers of metachromatic leukodystrophy.
Molecular genetics and metabolism 2003;79(2):83-90.
2003: David A Wenger; Stephanie Coppola; Shu-Ling Liu
Insights into the diagnosis and treatment of lysosomal storage diseases.
Archives of neurology 2003;60(3):322-8.
2002: David A Wenger; Stephanie Coppola; Shu-Ling Liu
Lysosomal storage disorders: diagnostic dilemmas and prospects for therapy.
Genetics in medicine : official journal of the American College of Medical Genetics 2002;4(6):412-9.
2002: Mario Sabatelli; Loreta Quaranta; Francesca Madia; G Lippi; A Conte; M Lo Monaco; Girolamo Di Trapani; Mohammad A Rafi; David A Wenger; A M Vaccaro; Pietro Tonali
Peripheral neuropathy with hypomyelinating features in adult-onset Krabbe's disease.
Neuromuscular disorders : NMD 2002;12(4):386-91.
2001: Kyra L Somers; DE Brown; R Fulton; Patricia C Schultheiss; Dwayne W Hamar; MO Smith; R Allison; Heather E Connally; C Just; TW Mitchell; David A Wenger; Mary Anna Thrall
Effects of dietary cholesterol restriction in a feline model of Niemann-Pick type C disease.
Journal of inherited metabolic disease 2001;24(4):427-36.
2001: Alice Luddi; M Volterrani; Michelina Strazza; A Smorlesi; Mohammad A Rafi; J Datto; David A Wenger; Elvira Costantino-Ceccarini
Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction.
Neurobiology of disease 2001;8(4):600-10.
2001: Paola Luzi; Mohammad A Rafi; Mariam Zaka; Mark Curtis; marie T vanier; David A Wenger
Generation of a mouse with low galactocerebrosidase activity by gene targeting: a new model of globoid cell leukodystrophy (Krabbe disease).
Molecular genetics and metabolism 2001;73(3):211-23.
2001: Gilles Millat; C Marçais; C Tomasetto; Karim Chikh; AH Fensom; Klaus Harzer; David A Wenger; K Ohno; marie T vanier
Niemann-Pick C1 disease: correlations between NPC1 mutations, levels of NPC1 protein, and phenotypes emphasize the functional significance of the putative sterol-sensing domain and of the cysteine-rich luminal loop.
American journal of human genetics 2001;68(6):1373-85.
2000: Patricia C Schultheiss; S A Gardner; J M Owens; David A Wenger; Mary Anna Thrall
Mucopolysaccharidosis VII in a cat.
Veterinary pathology 2000;37(5):502-5.
2000: David A Wenger; Mohammad A Rafi; Paola Luzi; J Datto; Elvira Costantino-Ceccarini
Krabbe disease: genetic aspects and progress toward therapy.
Molecular genetics and metabolism 2000;70(1):1-9.
2000: Nicola de Stefano; Maria Teresa Dotti; Marzia Mortilla; E Pappagallo; Paola Luzi; Mohammad A Rafi; Patrizia Formichi; D Inzitari; David A Wenger; Antonio Federico
Evidence of diffuse brain pathology and unspecific genetic characterization in a patient with an atypical form of adult-onset Krabbe disease.
Journal of neurology 2000;247(3):226-8.
2000: Joseph McGowan; Mark E Haskins; David A Wenger; Charles H Vite
Investigating demyelination in the brain in a canine model of globoid cell leukodystrophy (Krabbe disease) using magnetization transfer contrast: preliminary results.
Journal of computer assisted tomography 2000;24(2):316-21.
1999: Gilles Millat; C Marçais; Mohammad A Rafi; T Yamamoto; JA Morris; Peter G Pentchev; K Ohno; David A Wenger; marie T vanier
Niemann-Pick C1 disease: the I1061T substitution is a frequent mutant allele in patients of Western European descent and correlates with a classic juvenile phenotype.
American journal of human genetics 1999;65(5):1321-9.
1999: Laura B Jardim; Roberto Giugliani; Ricardo F Pires; S Haussen; Maira G Burin; Mohammad A Rafi; David A Wenger
Protracted course of Krabbe disease in an adult patient bearing a novel mutation.
Archives of neurology 1999;56(8):1014-7.
1999: Kyra L Somers; David A Wenger; Michael A Royals; Eugene D Carstea; Heather E Connally; T Kelly; R Kimball; Mary Anna Thrall
Complementation studies in human and feline Niemann-Pick type C disease.
Molecular genetics and metabolism 1999;66(2):117-21.
1999: Elvira Costantino-Ceccarini; Alice Luddi; M Volterrani; Michelina Strazza; Mohammad A Rafi; David A Wenger
Transduction of cultured oligodendrocytes from normal and twitcher mice by a retroviral vector containing human galactocerebrosidase (GALC) cDNA.
Neurochemical research 1999;24(2):287-93.
1999: David A Wenger; T Victoria; Mohammad A Rafi; Paola Luzi; marie T vanier; Charles H Vite; D F Patterson; M H Haskins
Globoid cell leukodystrophy in cairn and West Highland white terriers.
The Journal of heredity 1999;90(1):138-42.
1998: Gary B Baskin; Marion S Ratterree; Billie B Davison; KP Falkenstein; M R Clarke; John D England; marie T vanier; Paola Luzi; Mohammad A Rafi; David A Wenger
Genetic galactocerebrosidase deficiency (globoid cell leukodystrophy, Krabbe disease) in rhesus monkeys (Macaca mulatta).
Laboratory animal science 1998;48(5):476-82.
1998: F Cozzi; Charles H Vite; David A Wenger; T Victoria; Mark E Haskins
MRI and electrophysiological abnormalities in a case of canine globoid cell leucodystrophy.
The Journal of small animal practice 1998;39(8):401-5.
1997: Paola Luzi; T Victoria; Mohammad A Rafi; David A Wenger
Analysis of the 5' flanking region of the human galactocerebrosidase (GALC) gene.
Biochemical and molecular medicine 1997;62(2):159-64.
1997: Wim J Kleijer; J L M Keulemans; M van der Kraan; G G Geilen; R M van der Helm; Mohammad A Rafi; Paola Luzi; David A Wenger; D J J Halley; Otto P van Diggelen
Prevalent mutations in the GALC gene of patients with Krabbe disease of Dutch and other European origin.
Journal of inherited metabolic disease 1997;20(4):587-94.
1997: S M Dial; T Byrne; Mark E Haskins; P W Gasper; B Rose; David A Wenger; Mary Anna Thrall
Urine glycosaminoglycan concentrations in mucopolysaccharidosis VI-affected cats following bone marrow transplantation or leukocyte infusion.
Clinica chimica acta; international journal of clinical chemistry 1997;263(1):1-14.
1997: Paola Luzi; Mohammad A Rafi; T Victoria; Gary B Baskin; David A Wenger
Characterization of the rhesus monkey galactocerebrosidase (GALC) cDNA and gene and identification of the mutation causing globoid cell leukodystrophy (Krabbe disease) in this primate.
Genomics 1997;42(2):319-24.
1997: David A Wenger; Mohammad A Rafi; Paola Luzi
Molecular genetics of Krabbe disease (globoid cell leukodystrophy): diagnostic and clinical implications.
Human mutation 1997;10(4):268-79.
1996: Mohammad A Rafi; J Fugaro; S Amini; Paola Luzi; G de Gala; T Victoria; C Dubell; M Shahinfar; David A Wenger
Retroviral vector-mediated transfer of the galactocerebrosidase (GALC) cDNA leads to overexpression and transfer of GALC activity to neighboring cells.
Biochemical and molecular medicine 1996;58(2):142-50.
1996: Paola Luzi; Mohammad A Rafi; David A Wenger
Multiple mutations in the GALC gene in a patient with adult-onset Krabbe disease.
Annals of neurology 1996;40(1):116-9.
1996: T Victoria; Mohammad A Rafi; David A Wenger
Cloning of the canine GALC cDNA and identification of the mutation causing globoid cell leukodystrophy in West Highland White and Cairn terriers.
Genomics 1996;33(3):457-62.
1996: Mohammad A Rafi; Paola Luzi; J Zlotogora; David A Wenger
Two different mutations are responsible for Krabbe disease in the Druze and Moslem Arab populations in Israel.
Human genetics 1996;97(3):304-8.
1995: Mohammad A Rafi; Paola Luzi; Y Q Chen; David A Wenger
A large deletion together with a point mutation in the GALC gene is a common mutant allele in patients with infantile Krabbe disease.
Human molecular genetics 1995;4(8):1285-9.
1995: Paola Luzi; Mohammad A Rafi; David A Wenger
Structure and organization of the human galactocerebrosidase (GALC) gene.
Genomics 1995;26(2):407-9.
1995: N M Pastor-Soler; E M Schertz; Mohammad A Rafi; G de Gala; David A Wenger
Metachromatic leukodystrophy among southern Alaskan Eskimos: molecular and genetic studies.
Journal of inherited metabolic disease 1995;18(3):326-32.
1994: M Shahinfar; David A Wenger
Adult and infantile Gaucher disease in one family: mutational studies and clinical update.
The Journal of pediatrics 1994;125(6 Pt 1):919-21.
1994: S Chakraborty; Mohammad A Rafi; David A Wenger
Mutations in the lysosomal beta-galactosidase gene that cause the adult form of GM1 gangliosidosis.
American journal of human genetics 1994;54(6):1004-13.
1994: N M Pastor-Soler; Mohammad A Rafi; J D Hoffman; D Hu; David A Wenger
Metachromatic leukodystrophy in the Navajo Indian population: a splice site mutation in intron 4 of the arylsulfatase A gene.
Human mutation 1994;4(3):199-207.
1993: R Oehlmann; J Zlotogora; David A Wenger; R G Knowlton
Localization of the Krabbe disease gene (GALC) on chromosome 14 by multipoint linkage analysis.
American journal of human genetics 1993;53(6):1250-5.
1993: Y Q Chen; Mohammad A Rafi; G de Gala; David A Wenger
Cloning and expression of cDNA encoding human galactocerebrosidase, the enzyme deficient in globoid cell leukodystrophy.
Human molecular genetics 1993;2(11):1841-5.
1993: Y Q Chen; David A Wenger
Galactocerebrosidase from human urine: purification and partial characterization.
Biochimica et biophysica acta 1993;1170(1):53-61.
1993: Mohammad A Rafi; G de Gala; X L Zhang; David A Wenger
Mutational analysis in a patient with a variant form of Gaucher disease caused by SAP-2 deficiency.
Somatic cell and molecular genetics 1993;19(1):1-7.
1993: David A Wenger
Research update on lysosomal disorders with special emphasis on metachromatic leukodystrophy and Krabbe disease.
APMIS. Supplementum 1993;40():81-7.
1992: Mary C Phelan; G R Thomas; Robert A. Saul; R. Curtis Rogers; H A Taylor; David A Wenger; H E McDermid
Cytogenetic, biochemical, and molecular analyses of a 22q13 deletion.
American journal of medical genetics 1992;43(5):872-6.
1992: Mohammad A Rafi; S Amini; X L Zhang; David A Wenger
Correction of sulfatide metabolism after transfer of prosaposin cDNA to cultured cells from a patient with SAP-1 deficiency.
American journal of human genetics 1992;50(6):1252-8.
1991: X L Zhang; Mohammad A Rafi; G DeGala; David A Wenger
The mechanism for a 33-nucleotide insertion in mRNA causing sphingolipid activator protein (SAP-1)-deficient metachromatic leukodystrophy.
Human genetics 1991;87(2):211-5.
1991: David A Wenger; E Louie
Pseudodeficiencies of arylsulfatase A and galactocerebrosidase activities.
Developmental neuroscience 1991;13(4-5):216-21.
1990: J Zlotogora; S Chakraborty; R G Knowlton; David A Wenger
Krabbe disease locus mapped to chromosome 14 by genetic linkage.
American journal of human genetics 1990;47(1):37-44.
1990: X L Zhang; Mohammad A Rafi; G DeGala; David A Wenger
Insertion in the mRNA of a metachromatic leukodystrophy patient with sphingolipid activator protein-1 deficiency.
Proceedings of the National Academy of Sciences of the United States of America 1990;87(4):1426-30.
1990: Mohammad A Rafi; X L Zhang; G DeGala; David A Wenger
Detection of a point mutation in sphingolipid activator protein-1 mRNA in patients with a variant form of metachromatic leukodystrophy.
Biochemical and biophysical research communications 1990;166(2):1017-23.
1989: David A Wenger; G DeGala; C Williams; H A Taylor; Roger E Stevenson; J R Pruitt; J Miller; P D Garen; J D Balentine
Clinical, pathological, and biochemical studies on an infantile case of sulfatide/GM1 activator protein deficiency.
American journal of medical genetics 1989;33(2):255-65.