Tissue-specific variation in nonsense mutant transcript level and drug-induced read-through efficiency in the Cln1(R151X) mouse model of INCL
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Nonsense Suppression as an Approach to Treat Lysosomal Storage Diseases.Small molecules as therapeutic agents for inborn errors of metabolism.Homozygous PPT1 Splice Donor Mutation in a Cane Corso Dog With Neuronal Ceroid LipofuscinosisThe effect of PTC124 on choroideremia fibroblasts and iPSC-derived RPE raises considerations for therapy.
P2860
Tissue-specific variation in nonsense mutant transcript level and drug-induced read-through efficiency in the Cln1(R151X) mouse model of INCL
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2015 nî lūn-bûn
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2015年の論文
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2015年論文
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name
Tissue-specific variation in n ...... ln1(R151X) mouse model of INCL
@ast
Tissue-specific variation in n ...... ln1(R151X) mouse model of INCL
@en
type
label
Tissue-specific variation in n ...... ln1(R151X) mouse model of INCL
@ast
Tissue-specific variation in n ...... ln1(R151X) mouse model of INCL
@en
prefLabel
Tissue-specific variation in n ...... ln1(R151X) mouse model of INCL
@ast
Tissue-specific variation in n ...... ln1(R151X) mouse model of INCL
@en
P2093
P2860
P356
P1476
Tissue-specific variation in n ...... ln1(R151X) mouse model of INCL
@en
P2093
Attila D Kovács
David A Pearce
Vaughn Thada
P2860
P304
P356
10.1111/JCMM.12744
P577
2015-12-09T00:00:00Z