Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model

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Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model

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作者：Christian Hinderer^a ; Peter Bell^a ; Jean-Pierre Louboutin^b ; Nathan Katz^a ; Yanqing Zhu^a ; Gloria Lin^a ; Ruth Choa^a ; Jessica Bagel^c ; Patricia O'Donnell^c ; Caitlin A. Fitzgerald^c ; Therese Langan^c ; Ping Wang^c ; Margret L. Casal^c ; Mark E. Haskins^c ; James M. Wilson^a ; ^{wilsonjm@mail.med.upenn.edu" class="auth_mail" title="E-mail the corresponding author}
关键词：AAV ; Gene therapy ; Intrathecal ; MPS I ; Neonatal tolerance
刊名：Molecular Genetics and Metabolism
出版年：2016
出版时间：September-October 2016
年：2016
卷：119
期：1-2
页码：124-130
全文大小：913 K

文摘

class="label">•: MPS I dogs treated with gene therapy develop immune response against human IDUA.
class="label">•: MPS I dogs exposed to human IDUA as newborns develop tolerance to the protein.
class="label">•: Tolerance induction allows for accurate evaluation of human vector in canine model.
class="label">•: Important approach for preclinical evaluation of gene or protein therapeutics
class="label">•: Potential to induce tolerance to therapeutic proteins for recessive diseases

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