window.dataLayer = window.dataLayer || []; function gtag(){dataLayer.push(arguments);} gtag('js', new Date()); gtag('config', 'UA-48293608-1');
Logo-bi
BioImpacts. 2021;11(2): 135-146. doi: 10.34172/bi.2021.21
PMID: 33842284        PMCID: PMC8022232

Original Research

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

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

Cited by CrossRef: 13

1- 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]
2- Li Y, Dong X, Guo J, Lv Y. Gene therapy for Krabbe disease: evidence from mouse and canine models. Gene. 2026;980:149949 [Crossref]
3- Lin D, Ho C, Huang Y, Lee T, Huang Z, Wang T, Cheng W, Huang S. Durable Global Correction of CNS and PNS and Lifespan Rescue in Murine Globoid Cell Leukodystrophy via AAV9-Mediated Monotherapy. Cells. 2025;14(24):1942 [Crossref]
4- 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]
5- LeVine S, Tsau S. Substrate Reduction Therapy for Krabbe Disease: Exploring the Repurposing of the Antibiotic D-Cycloserine. Front Pediatr. 2022;9 [Crossref]
6- 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]
7- 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]
8- 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]
9- Wenger D, Luzi P, Rafi M. Advances in the Diagnosis and Treatment of Krabbe Disease. IJNS. 2021;7(3):57 [Crossref]
10- 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]
11- 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]
12- Nowacki J, Fields A, Fu M. Emerging cellular themes in leukodystrophies. Front Cell Dev Biol. 2022;10 [Crossref]
13- Maghazachi A. Globoid Cell Leukodystrophy (Krabbe Disease): An Update. ITT. 2023;Volume 12:105 [Crossref]

Poster

As a peer-reviewed international open-access journal, BioImpacts publishes articles on basic and translational aspects of pharmaceutical and biomedical sciences. 
Acceptance rate (2024): 20% 
Publication fee: Free of charge

Indexing/Abstracting Info
PubMed; PubMed Central; Scopus; Science Citation Index Expanded; Google Scholar;   SJR; Essential Science Indicators; Embase; EBSCOhost; CAS: DOAJ: SHERPA/RoMEO
Member of   
Follower of ICMJE
Permission: Creative Commons