Mohammad Rafi

TitleProfessor
InstitutionThomas Jefferson University
DepartmentNeurology
Address900 Walnut Street
Philadelphia PA 19107
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    1. Safary A, Moghaddas-Sani H, Akbarzadeh-Khiavi M, Khabbazzi A, Rafi MA, Omidi Y. Enzyme replacement combinational therapy: effective treatments for mucopolysaccharidoses. Expert Opin Biol Ther. 2021 Mar 11; 1-17. PMID: 33653197.
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    2. Rafi MA, Luzi P, Wenger DA. Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT? Bioimpacts. 2021; 11(2):135-146. PMID: 33842284.
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    3. Ilghami R, Mohammadhasanzadeh H, Barar J, Rafi MA. BioImpacts: An emerging global journal. Bioimpacts. 2020; 10(4):207-208. PMID: 32983935.
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    4. Rafi MA, Luzi P, Wenger DA. Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease. Bioimpacts. 2020; 10(2):105-115. PMID: 32363154.
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    5. Parvizpour S, Pourseif MM, Razmara J, Rafi MA, Omidi Y. Epitope-based vaccine design: a comprehensive overview of bioinformatics approaches. Drug Discov Today. 2020 Mar 20. PMID: 32205198.
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    6. Alizadeh S, Esmaeili A, Barzegari A, Rafi MA, Omidi Y. Bioengineered smart bacterial carriers for combinational targeted therapy of solid tumours. J Drug Target. 2020 Mar 16; 1-14. PMID: 32116051.
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    7. Vosoughi A, Sadigh-Eteghad S, Ghorbani M, Shahmorad S, Farhoudi M, Rafi MA, Omidi Y. Mathematical Models to Shed Light on Amyloid-Beta and Tau Protein Dependent Pathologies in Alzheimer's Disease. Neuroscience. 2019 Nov 01. PMID: 31682825.
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    8. Jafari B, Pourseif MM, Barar J, Rafi MA, Omidi Y. Peptide-mediated drug delivery across the blood-brain barrier for targeting brain tumors. Expert Opin Drug Deliv. 2019 06; 16(6):583-605. PMID: 31107110.
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    9. Safary A, Akbarzadeh Khiavi M, Omidi Y, Rafi MA. Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis. Cell Mol Life Sci. 2019 Sep; 76(17):3363-3381. PMID: 31101939.
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    10. Safary A, Akbarzadeh Khiavi M, Mousavi R, Barar J, Rafi MA. Enzyme replacement therapies: what is the best option? Bioimpacts. 2018; 8(3):153-157. PMID: 30211074.
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    11. Zamanlu M, Eskandani M, Mohammadian R, Entekhabi N, Rafi M, Farhoudi M. Spectrophotometric analysis of thrombolytic activity: SATA assay. Bioimpacts. 2018; 8(1):31-38. PMID: 29713600.
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    12. Rafi MA, Alavi A. Debate on human aging and lifespan. Bioimpacts. 2017; 7(3):135-137. PMID: 29159140.
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    13. Zamanlu M, Farhoudi M, Eskandani M, Mahmoudi J, Barar J, Rafi M, Omidi Y. Recent advances in targeted delivery of tissue plasminogen activator for enhanced thrombolysis in ischemic stroke. J Drug Target. 2017 Aug 10; 1-42. PMID: 28796540.
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    14. Barar J, Rafi MA, Pourseif MM, Omidi Y. Blood-brain barrier transport machineries and targeted therapy of brain diseases. Bioimpacts. 2016; 6(4):225-248. PMID: 28265539.
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    15. Wenger DA, Rafi MA, Luzi P. Krabbe disease: One Hundred years from the bedside to the bench to the bedside. J Neurosci Res. 2016 Nov; 94(11):982-9. PMID: 27638583.
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    16. Rafi MA. Gene therapy for CNS diseases - Krabbe disease. Bioimpacts. 2016; 6(2):69-70. PMID: 27525222.
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    17. Rafi MA, Omidi Y. A prospective highlight on exosomal nanoshuttles and cancer immunotherapy and vaccination. Bioimpacts. 2015; 5(3):117-22. PMID: 26457248.
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    18. Rafi MA, Rao HZ, Luzi P, Wenger DA. Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice. Mol Ther. 2015 Nov; 23(11):1681-90. PMID: 26329589.
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    19. Rafi MA. To impact or not to impact, this is not a question for BioImpacts! Bioimpacts. 2015; 5(1):1-2. PMID: 25901290.
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    20. Rafi MA, Rao HZ, Luzi P, Luddi A, Curtis MT, Wenger DA. Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features. Mol Genet Metab. 2015 Mar; 114(3):459-66. PMID: 25533112.
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    21. Acharya AP, Rafi M, Woods EC, Gardner AB, Murthy N. Metabolic engineering of lactate dehydrogenase rescues mice from acidosis. Sci Rep. 2014; 4:5189. PMID: 24898534.
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    22. Wenger DA, Luzi P, Rafi MA. Krabbe disease: Are certain mutations disease-causing only when specific polymorphisms are present or when inherited in trans with specific second mutations? Mol Genet Metab. 2014 Mar; 111(3):307-8. PMID: 24388568.
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    23. Wenger DA, Luzi P, Rafi MA. Lysosomal storage diseases: heterogeneous group of disorders. Bioimpacts. 2013; 3(4):145-7. PMID: 24455477.
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    24. Luzi P, Rafi MA, Rao HZ, Wenger DA. Sixteen novel mutations in the arylsulfatase A gene causing metachromatic leukodystrophy. Gene. 2013 Nov 10; 530(2):323-8. PMID: 24001781.
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    25. Rafi MA, Rao HZ, Luzi P, Curtis MT, Wenger DA. Extended Normal Life After AAVrh10-mediated Gene Therapy in the Mouse Model of Krabbe Disease. Mol Ther. 2012 Nov; 20(11):2031-42. PMID: 22850681.
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    26. Rafi MA. Gene and stem cell therapy: alone or in combination? Bioimpacts. 2011; 1(4):213-8. PMID: 23678430.
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    27. Acheampong EA, Roschel C, Mukhtar M, Srinivasan A, Rafi M, Pomerantz RJ, Parveen Z. Combined effects of hyperglycemic conditions and HIV-1 Nef: a potential model for induced HIV neuropathogenesis. Virol J. 2009; 6:183. PMID: 19878567.
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    28. Luzi P, Abraham RM, Rafi MA, Curtis M, Hooper DC, Wenger DA. Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy. Brain Res. 2009 Dec 1; 1300:146-58. PMID: 19748497.
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    29. Strazza M, Luddi A, Carbone M, Rafi MA, Costantino-Ceccarini E, Wenger DA. Significant correction of pathology in brains of twitcher mice following injection of genetically modified mouse neural progenitor cells. Mol Genet Metab. 2009 May; 97(1):27-34. PMID: 19217332.
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    30. Lissens W, Arena A, Seneca S, Rafi M, Sorge G, Liebaers I, Wenger D, Fiumara A. A single mutation in the GALC gene is responsible for the majority of late onset Krabbe disease patients in the Catania (Sicily, Italy) region. Hum Mutat. 2007 Jul; 28(7):742. PMID: 17579360.
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    31. Cheng X, Cheng X, Mukhtar M, Acheampong EA, Srinivasan A, Rafi M, Pomerantz RJ, Parveen Z. HIV-1 Vpr potently induces programmed cell death in the CNS in vivo. DNA Cell Biol. 2007 Feb; 26(2):116-31. PMID: 17328670.
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    32. Zaka M, Rafi MA, Rao HZ, Luzi P, Wenger DA. Insulin-like growth factor-1 provides protection against psychosine-induced apoptosis in cultured mouse oligodendrocyte progenitor cells using primarily the PI3K/Akt pathway. Mol Cell Neurosci. 2005 Nov; 30(3):398-407. PMID: 16169744.
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    33. Lin D, Fantz CR, Levy B, Rafi MA, Vogler C, Wenger DA, Sands MS. AAV2/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2. Mol Ther. 2005 Sep; 12(3):422-30. PMID: 15996520.
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    34. Luzi P, Rafi MA, Zaka M, Rao HZ, Curtis M, Vanier MT, Wenger DA. Biochemical and pathological evaluation of long-lived mice with globoid cell leukodystrophy after bone marrow transplantation. Mol Genet Metab. 2005 Sep-Oct; 86(1-2):150-9. PMID: 16169269.
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    35. Rafi MA, Zhi Rao H, Passini MA, Curtis M, Vanier MT, Zaka M, Luzi P, Wolfe JH, Wenger DA. AAV-mediated expression of galactocerebrosidase in brain results in attenuated symptoms and extended life span in murine models of globoid cell leukodystrophy. Mol Ther. 2005 May; 11(5):734-44. PMID: 15851012.
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    36. Luzi P, Zaka M, Rao HZ, Curtis M, Rafi MA, Wenger DA. 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. Neurochem Res. 2004 May; 29(5):881-9. PMID: 15139287.
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    37. Parveen Z, Mukhtar M, Rafi M, Wenger DA, Siddiqui KM, Siler CA, Dietzschold B, Pomerantz RJ, Schnell MJ, Dornburg R. Cell-type-specific gene delivery into neuronal cells in vitro and in vivo. Virology. 2003 Sep 15; 314(1):74-83. PMID: 14517061.
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    38. Rafi MA, Coppola S, Liu SL, Rao HZ, Wenger DA. Disease-causing mutations in cis with the common arylsulfatase A pseudodeficiency allele compound the difficulties in accurately identifying patients and carriers of metachromatic leukodystrophy. Mol Genet Metab. 2003 Jun; 79(2):83-90. PMID: 12809637.
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    39. Somers KL, Royals MA, Carstea ED, Rafi MA, Wenger DA, Thrall MA. Mutation analysis of feline Niemann-Pick C1 disease. Mol Genet Metab. 2003 Jun; 79(2):99-103. PMID: 12809639.
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    40. Sabatelli M, Quaranta L, Madia F, Lippi G, Conte A, Lo Monaco M, Di Trapani G, Rafi MA, Wenger DA, Vaccaro AM, Tonali P. Peripheral neuropathy with hypomyelinating features in adult-onset Krabbe's disease. Neuromuscul Disord. 2002 May; 12(4):386-91. PMID: 12062257.
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    41. Luddi A, Volterrani M, Strazza M, Smorlesi A, Rafi MA, Datto J, Wenger DA, Costantino-Ceccarini E. Retrovirus-mediated gene transfer and galactocerebrosidase uptake into twitcher glial cells results in appropriate localization and phenotype correction. Neurobiol Dis. 2001 Aug; 8(4):600-10. PMID: 11493025.
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    42. Luzi P, Rafi MA, Zaka M, Curtis M, Vanier MT, Wenger DA. Generation of a mouse with low galactocerebrosidase activity by gene targeting: a new model of globoid cell leukodystrophy (Krabbe disease). Mol Genet Metab. 2001 Jul; 73(3):211-23. PMID: 11461188.
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    43. Wenger DA, Rafi MA, Luzi P, Datto J, Costantino-Ceccarini E. Krabbe disease: genetic aspects and progress toward therapy. Mol Genet Metab. 2000 May; 70(1):1-9. PMID: 10833326.
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    44. De Stefano N, Dotti MT, Mortilla M, Pappagallo E, Luzi P, Rafi MA, Formichi P, Inzitari D, Wenger DA, Federico A. Evidence of diffuse brain pathology and unspecific genetic characterization in a patient with an atypical form of adult-onset Krabbe disease. J Neurol. 2000 Mar; 247(3):226-8. PMID: 10787123.
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    45. Millat G, Marçais C, Rafi MA, Yamamoto T, Morris JA, Pentchev PG, Ohno K, Wenger DA, Vanier MT. 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. Am J Hum Genet. 1999 Nov; 65(5):1321-9. PMID: 10521297.
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    46. Jardim LB, Giugliani R, Pires RF, Haussen S, Burin MG, Rafi MA, Wenger DA. Protracted course of Krabbe disease in an adult patient bearing a novel mutation. Arch Neurol. 1999 Aug; 56(8):1014-7. PMID: 10448809.
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    47. Costantino-Ceccarini E, Luddi A, Volterrani M, Strazza M, Rafi MA, Wenger DA. Transduction of cultured oligodendrocytes from normal and twitcher mice by a retroviral vector containing human galactocerebrosidase (GALC) cDNA. Neurochem Res. 1999 Feb; 24(2):287-93. PMID: 9972877.
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    48. Wenger DA, Victoria T, Rafi MA, Luzi P, Vanier MT, Vite C, Patterson DF, Haskins MH. Globoid cell leukodystrophy in cairn and West Highland white terriers. J Hered. 1999 Jan-Feb; 90(1):138-42. PMID: 9987921.
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    49. Baskin GB, Ratterree M, Davison BB, Falkenstein KP, Clarke MR, England JD, Vanier MT, Luzi P, Rafi MA, Wenger DA. Genetic galactocerebrosidase deficiency (globoid cell leukodystrophy, Krabbe disease) in rhesus monkeys (Macaca mulatta). Lab Anim Sci. 1998 Oct; 48(5):476-82. PMID: 10090061.
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    50. Luzi P, Victoria T, Rafi MA, Wenger DA. Analysis of the 5' flanking region of the human galactocerebrosidase (GALC) gene. Biochem Mol Med. 1997 Dec; 62(2):159-64. PMID: 9441867.
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    51. Kleijer WJ, Keulemans JL, van der Kraan M, Geilen GG, van der Helm RM, Rafi MA, Luzi P, Wenger DA, Halley DJ, van Diggelen OP. Prevalent mutations in the GALC gene of patients with Krabbe disease of Dutch and other European origin. J Inherit Metab Dis. 1997 Aug; 20(4):587-94. PMID: 9266397.
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    52. Luzi P, Rafi MA, Victoria T, Baskin GB, Wenger DA. 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 Jun 1; 42(2):319-24. PMID: 9192853.
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    53. Wenger DA, Rafi MA, Luzi P. Molecular genetics of Krabbe disease (globoid cell leukodystrophy): diagnostic and clinical implications. Hum Mutat. 1997; 10(4):268-79. PMID: 9338580.
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    54. Rafi MA, Fugaro J, Amini S, Luzi P, de Gala G, Victoria T, Dubell C, Shahinfar M, Wenger DA. Retroviral vector-mediated transfer of the galactocerebrosidase (GALC) cDNA leads to overexpression and transfer of GALC activity to neighboring cells. Biochem Mol Med. 1996 Aug; 58(2):142-50. PMID: 8812733.
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    55. Luzi P, Rafi MA, Wenger DA. Multiple mutations in the GALC gene in a patient with adult-onset Krabbe disease. Ann Neurol. 1996 Jul; 40(1):116-9. PMID: 8687180.
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    56. Victoria T, Rafi MA, Wenger DA. Cloning of the canine GALC cDNA and identification of the mutation causing globoid cell leukodystrophy in West Highland White and Cairn terriers. Genomics. 1996 May 1; 33(3):457-62. PMID: 8661004.
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    57. Rafi MA, Luzi P, Zlotogora J, Wenger DA. Two different mutations are responsible for Krabbe disease in the Druze and Moslem Arab populations in Israel. Hum Genet. 1996 Mar; 97(3):304-8. PMID: 8786069.
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    58. Luzi P, Rafi MA, Wenger DA. Characterization of the large deletion in the GALC gene found in patients with Krabbe disease. Hum Mol Genet. 1995 Dec; 4(12):2335-8. PMID: 8634707.
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    59. Rafi MA, Luzi P, Chen YQ, Wenger DA. A large deletion together with a point mutation in the GALC gene is a common mutant allele in patients with infantile Krabbe disease. Hum Mol Genet. 1995 Aug; 4(8):1285-9. PMID: 7581365.
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    60. Luzi P, Rafi MA, Wenger DA. Structure and organization of the human galactocerebrosidase (GALC) gene. Genomics. 1995 Mar 20; 26(2):407-9. PMID: 7601472.
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    61. Pastor-Soler NM, Schertz EM, Rafi MA, de Gala G, Wenger DA. Metachromatic leukodystrophy among southern Alaskan Eskimos: molecular and genetic studies. J Inherit Metab Dis. 1995; 18(3):326-32. PMID: 7474900.
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    62. Chakraborty S, Rafi MA, Wenger DA. Mutations in the lysosomal beta-galactosidase gene that cause the adult form of GM1 gangliosidosis. Am J Hum Genet. 1994 Jun; 54(6):1004-13. PMID: 8198123.
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    63. Cannizzaro LA, Chen YQ, Rafi MA, Wenger DA. Regional mapping of the human galactocerebrosidase gene (GALC) to 14q31 by in situ hybridization. Cytogenet Cell Genet. 1994; 66(4):244-5. PMID: 8162701.
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    64. Pastor-Soler NM, Rafi MA, Hoffman JD, Hu D, Wenger DA. Metachromatic leukodystrophy in the Navajo Indian population: a splice site mutation in intron 4 of the arylsulfatase A gene. Hum Mutat. 1994; 4(3):199-207. PMID: 7833949.
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    65. Chen YQ, Rafi MA, de Gala G, Wenger DA. Cloning and expression of cDNA encoding human galactocerebrosidase, the enzyme deficient in globoid cell leukodystrophy. Hum Mol Genet. 1993 Nov; 2(11):1841-5. PMID: 8281145.
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    66. Rafi MA, de Gala G, Zhang XL, Wenger DA. Mutational analysis in a patient with a variant form of Gaucher disease caused by SAP-2 deficiency. Somat Cell Mol Genet. 1993 Jan; 19(1):1-7. PMID: 8460394.
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    67. Rafi MA, Amini S, Zhang XL, Wenger DA. Correction of sulfatide metabolism after transfer of prosaposin cDNA to cultured cells from a patient with SAP-1 deficiency. Am J Hum Genet. 1992 Jun; 50(6):1252-8. PMID: 1350885.
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    68. Wenger DA, Williams C, Considine E and Rafi MA. Research developments on lysosomal disorders: metachromatic leukodystrophy and Gaucher disease. Int Pediatr. 1992; (7):34-39.
    69. Zhang XL, Rafi MA, DeGala G, Wenger DA. The mechanism for a 33-nucleotide insertion in mRNA causing sphingolipid activator protein (SAP-1)-deficient metachromatic leukodystrophy. Hum Genet. 1991 Jun; 87(2):211-5. PMID: 2066109.
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    70. Louie E, Rafi MA, Wenger DA. Leukocyte sonicates as a source for both enzyme assay and DNA amplification for mutational analysis of certain lysosomal disorders. Clin Chim Acta. 1991 May 31; 199(1):7-15. PMID: 1682071.
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    71. Zhang XL, Rafi MA, DeGala G, Wenger DA. Insertion in the mRNA of a metachromatic leukodystrophy patient with sphingolipid activator protein-1 deficiency. Proc Natl Acad Sci U S A. 1990 Feb; 87(4):1426-30. PMID: 1689485.
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    72. Rafi MA, Zhang XL, DeGala G, Wenger DA. Detection of a point mutation in sphingolipid activator protein-1 mRNA in patients with a variant form of metachromatic leukodystrophy. Biochem Biophys Res Commun. 1990 Jan 30; 166(2):1017-23. PMID: 2302219.
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    73. Rafi MA. Contribution to study of Artemisian Entomofaune in the south of France. Bull Soc Ent France. 1977; (82):113-118.
    74. Rafi A, Regard E, Beaumont A. [ Perfecting a simple method of organotypic thyroid gland culture in Xenopus laevis (Amphibia, Anoura)]. C R Acad Sci Hebd Seances Acad Sci D. 1976 Oct 27; 283(10):1229-32. PMID: 827362.
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    75. Bournier A and Rafi MA 5:166-169, 1975. Discovery of a Rare Species of Thysanopter (Ropotamothrips buresi). L'Entomologiste. 1975; (5):166-169.
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