Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
about
CNS-directed gene therapy for the treatment of neurologic and somatic mucopolysaccharidosis type II (Hunter syndrome)Gene therapy for lysosomal storage disorders: a good start.Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better?AAV Gene Therapy for MPS1-associated Corneal BlindnessNeonatal Immune Tolerance Induction to Allow Long-Term Studies With an Immunogenic Therapeutic Monoclonal Antibody in MiceA dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice.Adeno-associated viral gene therapy for mucopolysaccharidoses exhibiting neurodegeneration.Combination Therapies for Lysosomal Storage Diseases: A Complex Answer to a Simple Problem.Adeno-Associated Virus-Based Gene Therapy for CNS Diseases.Systemic delivery of adeno-associated viral vectors.Evaluation of intrathecal routes of administration for adeno-associated virus vectors in large animals.Prolonged Expression of Secreted Enzymes in Dogs After Liver-Directed Delivery of Sleeping Beauty Transposons: Implications for Non-Viral Gene Therapy of Systemic Disease.Human Bocavirus Type-1 Capsid Facilitates the Transduction of Ferret Airways by Adeno-Associated Virus Genomes.Intranasal Adeno-Associated Virus Mediated Gene Delivery and Expression of Human Iduronidase in the Central Nervous System: A Noninvasive and Effective Approach for Prevention of Neurologic Disease in Mucopolysaccharidosis Type I.Disease correction by AAV-mediated gene therapy in a new mouse model of mucopolysaccharidosis type IIID.Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model.Abnormal polyamine metabolism is unique to the neuropathic forms of MPS: potential for biomarker development and insight into pathogenesis.Phase I and II clinical trials for the mucopolysaccharidoses.AAV gene delivery to the spinal cord: serotypes, methods, candidate diseases, and clinical trials.
P2860
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P2860
Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates.
description
2015 nî lūn-bûn
@nan
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
2015年论文
@zh
2015年论文
@zh-cn
name
Neonatal Systemic AAV Induces ...... I Dogs and Nonhuman Primates.
@ast
Neonatal Systemic AAV Induces ...... I Dogs and Nonhuman Primates.
@en
type
label
Neonatal Systemic AAV Induces ...... I Dogs and Nonhuman Primates.
@ast
Neonatal Systemic AAV Induces ...... I Dogs and Nonhuman Primates.
@en
prefLabel
Neonatal Systemic AAV Induces ...... I Dogs and Nonhuman Primates.
@ast
Neonatal Systemic AAV Induces ...... I Dogs and Nonhuman Primates.
@en
P2093
P2860
P356
P1433
P1476
Neonatal Systemic AAV Induces ...... S I Dogs and Nonhuman Primates
@en
P2093
Alice F Tarantal
Christian Hinderer
Gloria Lin
Hongwei Yu
Jean-Pierre Louboutin
Jessica Bagel
Margret L Casal
Mark E Haskins
Patricia O'Donnell
Peter Bell
P2860
P304
P356
10.1038/MT.2015.99
P577
2015-05-29T00:00:00Z