Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an AAV vector expressing human SMN.
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Micro-dystrophin gene therapy goes systemic in Duchenne muscular dystrophy patients.Safety First: Perspective on Patient-Centered Development of AAV Gene Therapy Products.A Rationally Engineered Capsid Variant of AAV9 for Systemic CNS-Directed and Peripheral Tissue-Detargeted Gene Delivery in Neonates.Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX.Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular DystrophyLessons learned from lung and liver in-vivo gene therapy: implications for the futureShorter Phosphorodiamidate Morpholino Splice-Switching Oligonucleotides May Increase Exon-Skipping Efficacy in DMDNeuro-Immuno-Gene- and Genome-Editing-Therapy for Alzheimer's Disease: Are We There Yet?Dose-Dependent Effects of FKRP Gene-Replacement Therapy on Functional Rescue and Longevity in Dystrophic MiceNew Directions for SMA TherapyHerpes Simplex Virus Vectors for Gene Transfer to the Central Nervous System
P2860
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P2860
Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an AAV vector expressing human SMN.
description
2018 nî lūn-bûn
@nan
2018年の論文
@ja
2018年学术文章
@wuu
2018年学术文章
@zh
2018年学术文章
@zh-cn
2018年学术文章
@zh-hans
2018年学术文章
@zh-my
2018年学术文章
@zh-sg
2018年學術文章
@yue
2018年學術文章
@zh-hant
name
Severe toxicity in nonhuman pr ...... V vector expressing human SMN.
@en
Severe toxicity in nonhuman pr ...... V vector expressing human SMN.
@nl
type
label
Severe toxicity in nonhuman pr ...... V vector expressing human SMN.
@en
Severe toxicity in nonhuman pr ...... V vector expressing human SMN.
@nl
prefLabel
Severe toxicity in nonhuman pr ...... V vector expressing human SMN.
@en
Severe toxicity in nonhuman pr ...... V vector expressing human SMN.
@nl
P2093
P2860
P356
P1433
P1476
Severe toxicity in nonhuman pr ...... V vector expressing human SMN.
@en
P2093
Cecilia Dyer
Christian Hinderer
Elizabeth L Buza
Laura K Richman
Nathan Katz
Peter Bell
Tamara Goode
P2860
P304
P356
10.1089/HUM.2018.015
P577
2018-01-29T00:00:00Z