AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy

Бусад хувилбаруудыг харуулах (1)

Номзүйн дэлгэрэнгүй
Үндсэн зохиолчид:	Goyenvalle, A, Babbs, A, Garcia, L, Davies, K
Формат:	Conference item
Хэвлэсэн:	2010

Ижил төстэй зүйлс

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
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Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
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Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
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AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy
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Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.
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New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy.
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The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery
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Possibility of exon skipping therapy for Duchenne muscular dystrophy in Russian patients: present and future
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Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
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Newborn screening of duchenne muscular dystrophy specifically targeting deletions amenable to exon-skipping therapy
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In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.
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Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
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NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy
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In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy.
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From diagnosis to therapy in Duchenne muscular dystrophy
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Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy.
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U1 snRNA interactions with deep intronic sequences regulate splicing of multiple exons of spinal muscular atrophy genes
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