Hematopoietic Stem cell transplantation and lentiviral vector‐based gene therapy for Krabbe's disease: Present convictions and future prospects

Currently, presymtomatic hematopoietic stem and progenitor cell transplantation (HSPCT) is the only therapeutic modality that alleviates Krabbe's disease (KD)‐induced central nervous system damage. However, all HSPCT‐treated patients exhibit severe deterioration in peripheral nervous system fun...

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Bibliografiske detaljer
Udgivet i:J Neurosci Res
Main Authors: Hu, Peirong, Li, Yedda, Nikolaishvili‐Feinberg, Nana, Scesa, Giuseppe, Bi, Yanmin, Pan, Dao, Moore, Dominic, Bongarzone, Ernesto R., Sands, Mark S., Miller, Ryan, Kafri, Tal
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2016
Fag:
Therapies and Clinical Management
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5027985/
https://ncbi.nlm.nih.gov/pubmed/27638600
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jnr.23847
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