Can early treatment of twitcher mice with high dose AAVrh10-GALC eliminate the need for BMT?

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

Cited by CrossRef: 11

1- 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]

2- 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]

3- 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]

4- Wenger D, Luzi P, Rafi M. Advances in the Diagnosis and Treatment of Krabbe Disease. IJNS. 2021;7(3):57 [Crossref]

5- Metovic J, Li Y, Gong Y, Eichler F. Gene therapy for the leukodystrophies: From preclinical animal studies to clinical trials. Neurotherapeutics. 2024;21(4):e00443 [Crossref]

6- Evans L, Gawron J, Sim F, Feltri M, Marziali L, Signore G. Human iPSC-derived myelinating organoids and globoid cells to study Krabbe disease. PLoS ONE. 2024;19(12):e0314858 [Crossref]

7- Nowacki J, Fields A, Fu M. Emerging cellular themes in leukodystrophies. Front Cell Dev Biol. 2022;10 [Crossref]

8- Heller G, Bradbury A, Sands M, Bongarzone E. Preclinical studies in Krabbe disease: A model for the investigation of novel combination therapies for lysosomal storage diseases. Molecular Therapy. 2023;31(1):7 [Crossref]

9- LeVine S, Tsau S. Substrate Reduction Therapy for Krabbe Disease: Exploring the Repurposing of the Antibiotic D-Cycloserine. Front Pediatr. 2022;9 [Crossref]

10- Herdt A, Peng H, Dickson D, Golde T, Eckman E, Lee C. Brain Targeted AAV1-GALC Gene Therapy Reduces Psychosine and Extends Lifespan in a Mouse Model of Krabbe Disease. Genes. 2023;14(8):1517 [Crossref]

11- Maghazachi A. Globoid Cell Leukodystrophy (Krabbe Disease): An Update. ITT. 2023;Volume 12:105 [Crossref]

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