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

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

Εμφάνιση άλλων εκδόσεων (1)

Λεπτομέρειες βιβλιογραφικής εγγραφής
Κύριοι συγγραφείς:	Goyenvalle, A, Babbs, A, Garcia, L, Davies, K
Μορφή:	Conference item
Έκδοση:	2010

Παρόμοια τεκμήρια

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
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Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.
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Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy
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Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.
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Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
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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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Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice
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Antisense-induced exon skipping for duplications 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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New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy.
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Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors
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Assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy
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Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis
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The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery
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Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice
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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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Exon skipping peptide-pmos for correction of dystrophin in mouse models of duchenne muscular dystrophy
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Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy
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CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy
ανά: Harry Wilton-Clark, κ.ά.
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Newborn screening of duchenne muscular dystrophy specifically targeting deletions amenable to exon-skipping therapy
ανά: Pablo Beckers, κ.ά.
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Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy
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Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
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Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
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Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA
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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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Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy
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Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy.
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Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in 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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Correction: Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy 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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