AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy

AAV genome loss from dystrophic mouse muscles during AAV-U7 snRNA-mediated exon-skipping therapy

In the context of future adeno-associated viral (AAV)-based clinical trials for Duchenne myopathy, AAV genome fate in dystrophic muscles is of importance considering the viral capsid immunogenicity that prohibits recurring treatments. We showed that AAV genomes encoding non-therapeutic U7 were lost...

पूर्ण विवरण

ग्रंथसूची विवरण
मुख्य लेखकों:	Le Hir, M, Goyenvalle, A, Peccate, C, Précigout, G, Davies, K, Voit, T, Garcia, L, Lorain, S
स्वरूप:	Journal article
भाषा:	English
प्रकाशित:	2013

समान संसाधन

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2010)

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2011)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2012)

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice
द्वारा: Philippine Aupy, और अन्य
प्रकाशित: (2020-06-01)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2009)

Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2011)

Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice
द्वारा: Christina Gedicke‐Hornung, और अन्य
प्रकाशित: (2013-05-01)

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2009)

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2012)

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2012)

Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA
द्वारा: Paolo Peruzzo, और अन्य
प्रकाशित: (2025-02-01)

Use of antisense-mediated exon skipping to generate dystrophin in muscles of the mdx dystrophic mouse
द्वारा: Lu, Q, और अन्य
प्रकाशित: (2004)

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

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2010)

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.
द्वारा: Lu, Q, और अन्य
प्रकाशित: (2003)

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
द्वारा: Tabatha R. Simmons, और अन्य
प्रकाशित: (2021-06-01)

Exon exchange approach to repair Duchenne dystrophin transcripts.
द्वारा: Stéphanie Lorain, और अन्य
प्रकाशित: (2010-01-01)

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
द्वारा: Nicolas Wein, और अन्य
प्रकाशित: (2022-09-01)

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.
द्वारा: Eckenfelder, A, और अन्य
प्रकाशित: (2012)

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery
द्वारा: Agathe Eckenfelder, और अन्य
प्रकाशित: (2012-01-01)

Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) and AAV-U7 rescues the severe DMD phenotype in mice
द्वारा: Forand, A, और अन्य
प्रकाशित: (2020)

Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice
द्वारा: Anne Forand, और अन्य
प्रकाशित: (2020-06-01)

Exon Skipping in a Dysf-Missense Mutant Mouse Model
द्वारा: Jakub Malcher, और अन्य
प्रकाशित: (2018-12-01)

Nab Escaping AAV Mutants Isolated from Mouse Muscles
द्वारा: Zheng Chai, और अन्य
प्रकाशित: (2018-05-01)

AAV genome modification for efficient AAV production
द्वारा: Walaa Asaad, और अन्य
प्रकाशित: (2023-04-01)

U1 snRNA interactions with deep intronic sequences regulate splicing of multiple exons of spinal muscular atrophy genes
द्वारा: Eric W. Ottesen, और अन्य
प्रकाशित: (2024-07-01)

A single m6A modification in U6 snRNA diversifies exon sequence at the 5’ splice site
द्वारा: Yuma Ishigami, और अन्य
प्रकाशित: (2021-05-01)

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.
द्वारा: Aoki, Y, और अन्य
प्रकाशित: (2012)

Highly efficient CRISPR/Cas9‐mediated exon skipping for recessive dystrophic epidermolysis bullosa
द्वारा: Alex du Rand, और अन्य
प्रकाशित: (2024-07-01)

T Cell-Mediated Immune Responses to AAV and AAV Vectors
द्वारा: Hildegund C. J. Ertl
प्रकाशित: (2021-04-01)

Rational Design of AAV-rh74, AAV3B, and AAV8 with Limited Liver Targeting
द्वारा: Christopher Chan, और अन्य
प्रकाशित: (2023-10-01)

Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models
द्वारा: Amel Saoudi, और अन्य
प्रकाशित: (2023-03-01)

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.
द्वारा: Yokota, T, और अन्य
प्रकाशित: (2012)

Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa
द्वारा: Jeroen Bremer, और अन्य
प्रकाशित: (2016-01-01)

U1 snRNA associates with TFIIH and regulates transcriptional initiation.
द्वारा: Kwek, K, और अन्य
प्रकाशित: (2002)

Mechanism of U6 snRNA oligouridylation by human TUT1
द्वारा: Seisuke Yamashita, और अन्य
प्रकाशित: (2023-08-01)

Crystal structures of U6 snRNA-specific terminal uridylyltransferase
द्वारा: Seisuke Yamashita, और अन्य
प्रकाशित: (2017-06-01)

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
द्वारा: Goyenvalle, A, और अन्य
प्रकाशित: (2010)

AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery
द्वारा: Sanna Koponen, और अन्य
प्रकाशित: (2023-07-01)