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Bioimpacts. 2020;10(2): 105-115. doi: 10.34172/bi.2020.13
PMID: 32363154        PMCID: PMC7186542

Original Research

Conditions for combining gene therapy with bone marrow transplantation in murine Krabbe disease

Mohammad A. Rafi 1 ORCID, Paola Luzi 1, David A. Wenger 1 * ORCID

Cited by CrossRef: 19

1- Bradbury A, Ream M. Recent Advancements in the Diagnosis and Treatment of Leukodystrophies. Seminars in Pediatric Neurology. 2021;37:100876 [Crossref]
2- Bradbury A, Bongarzone E, Sands M. Krabbe disease: New hope for an old disease. Neuroscience Letters. 2021;752:135841 [Crossref]
3- von Jonquieres G, Rae C, Housley G. Emerging Concepts in Vector Development for Glial Gene Therapy: Implications for Leukodystrophies. Front Cell Neurosci. 2021;15 [Crossref]
4- Grabowski G, Mistry P. Therapies for lysosomal storage diseases: Principles, practice, and prospects for refinements based on evolving science. Molecular Genetics and Metabolism. 2022;137(1-2):81 [Crossref]
5- Sawamura M, Tachikawa K, Hikawa R, Akiyama H, Kaji S, Yasuda K, Leu A, Hong H, Mukthavaram R, Chivukula P, Yamakado H, Hirabayashi Y, Takahashi R, Matsuzawa S. A lipid nanoparticle-based oligodendrocyte-specific mRNA therapy. Molecular Therapy - Nucleic Acids. 2024;35(4):102380 [Crossref]
6- Hordeaux J, Jeffrey B, Jian J, Choudhury G, Michalson K, Mitchell T, Buza E, Chichester J, Dyer C, Bagel J, Vite C, Bradbury A, Wilson J. Efficacy and Safety of a Krabbe Disease Gene Therapy. Human Gene Therapy. 2022;33(9-10):499 [Crossref]
7- Zhou L, Wang Y, Xu Y, Zhang Y, Zhu C. Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders. Molecular Therapy - Methods & Clinical Development. 2024;32(4):101357 [Crossref]
8- Rafi M, Luzi P, Wenger D. Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?. Bioimpacts. 2021;11(2):135 [Crossref]
9- Wenger D, Luzi P, Rafi M. Advances in the Diagnosis and Treatment of Krabbe Disease. IJNS. 2021;7(3):57 [Crossref]
10- Nasir G, Chopra R, Elwood F, Ahmed S. Krabbe Disease: Prospects of Finding a Cure Using AAV Gene Therapy. Front Med. 2021;8 [Crossref]
11- Jensen T, Gøtzsche C, Woldbye D. Current and Future Prospects for Gene Therapy for Rare Genetic Diseases Affecting the Brain and Spinal Cord. Front Mol Neurosci. 2021;14 [Crossref]
12- Gupta A, Raymond G, Pierpont E, Kemp S, McIvor R, Rayannavar A, Miller B, Lund T, Orchard P. Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy. Expert Opinion on Biological Therapy. 2022;22(9):1151 [Crossref]
13- Jyoti P, Shakappa D. Glycosphingolipids Associated Metabolic Disorders. J Pharm Nutr Sci. 2024;14:16 [Crossref]
14- Ketata I, Ellouz E. From pathological mechanisms in Krabbe disease to cutting‐edge therapy: A comprehensive review. Neuropathology. 2024;44(4):255 [Crossref]
15- Shaimardanova A, Solovyeva V, Issa S, Rizvanov A. Gene Therapy of Sphingolipid Metabolic Disorders. IJMS. 2023;24(4):3627 [Crossref]
16- Feltri M, Weinstock N, Favret J, Dhimal N, Wrabetz L, Shin D. Mechanisms of demyelination and neurodegeneration in globoid cell leukodystrophy. Glia. 2021;69(10):2309 [Crossref]
17- Bradbury A, Bagel J, Swain G, Miyadera K, Pesayco J, Assenmacher C, Brisson B, Hendricks I, Wang X, Herbst Z, Pyne N, Odonnell P, Shelton G, Gelb M, Hackett N, Szabolcs P, Vite C, Escolar M. Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy. Molecular Therapy. 2024;32(1):44 [Crossref]

As a peer-reviewed international open-access journal, BioImpacts publishes articles on basic and translational aspects of pharmaceutical and biomedical sciences. 
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