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

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

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Xehetasun bibliografikoak
Egile Nagusiak:	Goyenvalle, A, Babbs, A, Garcia, L, Davies, K
Formatua:	Conference item
Argitaratua:	2010

Antzeko izenburuak

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
nork: Goyenvalle, A, et al.
Argitaratua: (2011)

Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.
nork: Goyenvalle, A, et al.
Argitaratua: (2011)

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy
nork: Goyenvalle, A, et al.
Argitaratua: (2012)

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.
nork: Goyenvalle, A, et al.
Argitaratua: (2012)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
nork: Goyenvalle, A, et al.
Argitaratua: (2009)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
nork: Goyenvalle, A, et al.
Argitaratua: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
nork: Goyenvalle, A, et al.
Argitaratua: (2012)

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy
nork: Le Hir, M, et al.
Argitaratua: (2013)

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.
nork: Goyenvalle, A, et al.
Argitaratua: (2009)

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice
nork: Philippine Aupy, et al.
Argitaratua: (2020-06-01)

Antisense-induced exon skipping for duplications in Duchenne muscular dystrophy
nork: van Ommen Gert-Jan B, et al.
Argitaratua: (2007-07-01)

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.
nork: Eckenfelder, A, et al.
Argitaratua: (2012)

New Approach for Antisense Oligonucleotide-Mediated Exon Skipping in Duchenne Muscular Dystrophy.
nork: Aoki, Y, et al.
Argitaratua: (2012)

Targeting Duchenne muscular dystrophy by skipping DMD exon 45 with base editors
nork: Michael Gapinske, et al.
Argitaratua: (2023-09-01)

Assessment of the feasibility of exon 45–55 multiexon skipping for duchenne muscular dystrophy
nork: van Ommen Gert-Jan B, et al.
Argitaratua: (2008-12-01)

Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis
nork: Yuko Shimizu-Motohashi, et al.
Argitaratua: (2018-06-01)

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery
nork: Agathe Eckenfelder, et al.
Argitaratua: (2012-01-01)

Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice
nork: Christina Gedicke‐Hornung, et al.
Argitaratua: (2013-05-01)

Possibility of exon skipping therapy for Duchenne muscular dystrophy in Russian patients: present and future
nork: E. V. Zinina, et al.
Argitaratua: (2024-09-01)

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
nork: Nicolas Wein, et al.
Argitaratua: (2022-09-01)

Exon skipping peptide-pmos for correction of dystrophin in mouse models of duchenne muscular dystrophy
nork: Betts, C, et al.
Argitaratua: (2014)

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy.
nork: Goyenvalle, A, et al.
Argitaratua: (2011)

Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy
nork: Aoki, Y, et al.
Argitaratua: (2011)

CRISPR-Cas9-mediated exon skipping as a cardioprotective strategy in Duchenne muscular dystrophy
nork: Harry Wilton-Clark, et al.
Argitaratua: (2023-09-01)

Newborn screening of duchenne muscular dystrophy specifically targeting deletions amenable to exon-skipping therapy
nork: Pablo Beckers, et al.
Argitaratua: (2021-02-01)

Upper limb disease evolution in exon 53 skipping eligible patients with Duchenne muscular dystrophy
nork: Charlotte Lilien, et al.
Argitaratua: (2021-10-01)

Evaluation of 2’-Deoxy-2’-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
nork: Silvana M G Jirka, et al.
Argitaratua: (2015-01-01)

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
nork: Tabatha R. Simmons, et al.
Argitaratua: (2021-06-01)

Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA
nork: Paolo Peruzzo, et al.
Argitaratua: (2025-02-01)

In vitro evaluation of novel antisense oligonucleotides is predictive of in vivo exon skipping activity for Duchenne muscular dystrophy.
nork: Wang, Q, et al.
Argitaratua: (2010)

Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy
nork: Goyenvalle Aurélie, et al.
Argitaratua: (2011-02-01)

Wild-type mouse models to screen antisense oligonucleotides for exon-skipping efficacy in Duchenne muscular dystrophy.
nork: Limin Cao, et al.
Argitaratua: (2014-01-01)

Novel Cationic Carotenoid Lipids as Delivery Vectors of Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy
nork: Vassilia Partali, et al.
Argitaratua: (2012-01-01)

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
nork: Liubov V. Gushchina, et al.
Argitaratua: (2023-12-01)

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy
nork: Naoki Watanabe, et al.
Argitaratua: (2018-12-01)

In silico screening based on predictive algorithms as a design tool for exon skipping oligonucleotides in Duchenne muscular dystrophy.
nork: Yusuke Echigoya, et al.
Argitaratua: (2015-01-01)

Correction: Correction: Wild-Type Mouse Models to Screen Antisense Oligonucleotides for Exon-Skipping Efficacy in Duchenne Muscular Dystrophy.
nork: PLOS ONE Staff
Argitaratua: (2019-01-01)

From diagnosis to therapy in Duchenne muscular dystrophy
nork: Babbs, A, et al.
Argitaratua: (2020)

Digital Droplet PCR for the Absolute Quantification of Exon Skipping Induced by Antisense Oligonucleotides in (Pre-)Clinical Development for Duchenne Muscular Dystrophy.
nork: Ruurd C Verheul, et al.
Argitaratua: (2016-01-01)

U1 snRNA interactions with deep intronic sequences regulate splicing of multiple exons of spinal muscular atrophy genes
nork: Eric W. Ottesen, et al.
Argitaratua: (2024-07-01)