Splicing-correcting therapeutic approaches for retinal dystrophies: where endogenous gene regulation and specificity matter.
about
Delivery is key: lessons learnt from developing splice-switching antisense therapiesUnravelling the Complexity of Inherited Retinal Dystrophies Molecular Testing: Added Value of Targeted Next-Generation Sequencing.Double-target Antisense U1snRNAs Correct Mis-splicing Due to c.639+861C>T and c.639+919G>A GLA Deep Intronic Mutations.RNA Biology in Retinal Development and Disease.
P2860
Splicing-correcting therapeutic approaches for retinal dystrophies: where endogenous gene regulation and specificity matter.
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article científic
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article scientifique
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articol științific
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articolo scientifico
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artigo científico
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artigo científico
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artículo científico
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Splicing-correcting therapeuti ...... lation and specificity matter.
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type
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Splicing-correcting therapeuti ...... lation and specificity matter.
@en
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Splicing-correcting therapeuti ...... lation and specificity matter.
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P356
P1476
Splicing-correcting therapeuti ...... lation and specificity matter.
@en
P2093
Niccolò Bacchi
Simona Casarosa
P304
P356
10.1167/IOVS.14-14544
P407
P577
2014-05-27T00:00:00Z