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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2016
Assuntos:
Therapies and Clinical Management
Acesso em linha: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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