PABPN1 gene therapy for oculopharyngeal muscular dystrophy
about
Dysfunctional transcripts are formed by alternative polyadenylation in OPMD.Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD.Novel mouse models of oculopharyngeal muscular dystrophy (OPMD) reveal early onset mitochondrial defects and suggest loss of PABPN1 may contribute to pathology.Ptosis in childhood: A clinical sign of several disorders: Case series reports and literature review
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PABPN1 gene therapy for oculopharyngeal muscular dystrophy
description
article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 31 March 2017
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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vědecký článek
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name
PABPN1 gene therapy for oculopharyngeal muscular dystrophy
@en
PABPN1 gene therapy for oculopharyngeal muscular dystrophy.
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type
label
PABPN1 gene therapy for oculopharyngeal muscular dystrophy
@en
PABPN1 gene therapy for oculopharyngeal muscular dystrophy.
@nl
prefLabel
PABPN1 gene therapy for oculopharyngeal muscular dystrophy
@en
PABPN1 gene therapy for oculopharyngeal muscular dystrophy.
@nl
P2093
P2860
P50
P356
P1476
PABPN1 gene therapy for oculopharyngeal muscular dystrophy
@en
P2093
H Bachtarzi
J Lacau St Guily
K Mamchaoui
M Polay Espinoza
P2860
P2888
P356
10.1038/NCOMMS14848
P407
P577
2017-03-31T00:00:00Z