Concepedia

Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy

Allison M. Bradbury, Jessica Bagel, Gary P. Swain, Keiko Miyadera, Jill P. Pesayco, Charles‐Antoine Assenmacher, Becky K. Brisson, Ian J. Hendricks, Xiao H. Wang, Zackary M. Herbst,

Molecular Therapy · 2023 · 12 citations · 28 references

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Concepts

References

28

Transplantation of Umbilical-Cord Blood in Babies with Infantile Krabbe's Disease

Maria L. Escolar, Michele D. Poe, James M. Provenzale et al. · New England Journal of Medicine · 2005 · 713 citations · Full text

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Prolonged Survival and Remyelination After Hematopoietic Cell Transplantation in the Twitcher Mouse

Andrew M. Yeager, Susan Brennan, Carol W. Tiffany et al. · Science · 1984 · 171 citations

, , +17

Newborn screening for Krabbe disease in New York State: the first eight years’ experience

Joseph J. Orsini, Denise M. Kay, Carlos A. Saavedra‐Matiz et al. · Genetics in Medicine · 2016 · 111 citations · Full text

, , +13

Developmental outcomes of cord blood transplantation for Krabbe disease

Matthew D. Wright, Michele D. Poe, Anthony DeRenzo et al. · Neurology · 2017 · 102 citations · Full text

, , +17

Clinical outcomes of children with abnormal newborn screening results for Krabbe disease in New York State

Melissa Wasserstein, Mary R. Andriola, Georgianne L. Arnold et al. · Genetics in Medicine · 2016 · 99 citations · Full text

, , +13