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...

Detaylı Bibliyografya
Asıl Yazarlar:	Le Hir, M, Goyenvalle, A, Peccate, C, Précigout, G, Davies, K, Voit, T, Garcia, L, Lorain, S
Materyal Türü:	Journal article
Dil:	English
Baskı/Yayın Bilgisi:	2013

Benzer Materyaller

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2010)

AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2011)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2012)

Long-Term Efficacy of AAV9-U7snRNA-Mediated Exon 51 Skipping in mdx52 Mice
Yazar:: Philippine Aupy, ve diğerleri
Baskı/Yayın Bilgisi: (2020-06-01)

Upgrading U7snRNA To Complete Efficient Rescue of Dystrophin by Exon-Skipping in DMD Patients
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2009)

Engineering exon-skipping vectors expressing U7 snRNA constructs for Duchenne muscular dystrophy gene therapy.
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2011)

Rescue of cardiomyopathy through U7snRNA‐mediated exon skipping in Mybpc3‐targeted knock‐in mice
Yazar:: Christina Gedicke‐Hornung, ve diğerleri
Baskı/Yayın Bilgisi: (2013-05-01)

Enhanced exon-skipping induced by U7 snRNA carrying a splicing silencer sequence: Promising tool for DMD therapy.
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2009)

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2012)

Engineering multiple U7snRNA constructs to induce single and multiexon-skipping for Duchenne muscular dystrophy.
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2012)

Rescue of common and rare exon 2 skipping variants of the GAA gene using modified U1 snRNA
Yazar:: Paolo Peruzzo, ve diğerleri
Baskı/Yayın Bilgisi: (2025-02-01)

Use of antisense-mediated exon skipping to generate dystrophin in muscles of the mdx dystrophic mouse
Yazar:: Lu, Q, ve diğerleri
Baskı/Yayın Bilgisi: (2004)

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model
Yazar:: Liubov V. Gushchina, ve diğerleri
Baskı/Yayın Bilgisi: (2023-12-01)

Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2010)

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse.
Yazar:: Lu, Q, ve diğerleri
Baskı/Yayın Bilgisi: (2003)

Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping
Yazar:: Tabatha R. Simmons, ve diğerleri
Baskı/Yayın Bilgisi: (2021-06-01)

Exon exchange approach to repair Duchenne dystrophin transcripts.
Yazar:: Stéphanie Lorain, ve diğerleri
Baskı/Yayın Bilgisi: (2010-01-01)

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
Yazar:: Nicolas Wein, ve diğerleri
Baskı/Yayın Bilgisi: (2022-09-01)

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery.
Yazar:: Eckenfelder, A, ve diğerleri
Baskı/Yayın Bilgisi: (2012)

The Cellular Processing Capacity Limits the Amounts of Chimeric U7 snRNA Available for Antisense Delivery
Yazar:: Agathe Eckenfelder, ve diğerleri
Baskı/Yayın Bilgisi: (2012-01-01)

Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) and AAV-U7 rescues the severe DMD phenotype in mice
Yazar:: Forand, A, ve diğerleri
Baskı/Yayın Bilgisi: (2020)

Combined Treatment with Peptide-Conjugated Phosphorodiamidate Morpholino Oligomer-PPMO and AAV-U7 Rescues the Severe DMD Phenotype in Mice
Yazar:: Anne Forand, ve diğerleri
Baskı/Yayın Bilgisi: (2020-06-01)

Exon Skipping in a Dysf-Missense Mutant Mouse Model
Yazar:: Jakub Malcher, ve diğerleri
Baskı/Yayın Bilgisi: (2018-12-01)

Nab Escaping AAV Mutants Isolated from Mouse Muscles
Yazar:: Zheng Chai, ve diğerleri
Baskı/Yayın Bilgisi: (2018-05-01)

AAV genome modification for efficient AAV production
Yazar:: Walaa Asaad, ve diğerleri
Baskı/Yayın Bilgisi: (2023-04-01)

U1 snRNA interactions with deep intronic sequences regulate splicing of multiple exons of spinal muscular atrophy genes
Yazar:: Eric W. Ottesen, ve diğerleri
Baskı/Yayın Bilgisi: (2024-07-01)

A single m6A modification in U6 snRNA diversifies exon sequence at the 5’ splice site
Yazar:: Yuma Ishigami, ve diğerleri
Baskı/Yayın Bilgisi: (2021-05-01)

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.
Yazar:: Aoki, Y, ve diğerleri
Baskı/Yayın Bilgisi: (2012)

Highly efficient CRISPR/Cas9‐mediated exon skipping for recessive dystrophic epidermolysis bullosa
Yazar:: Alex du Rand, ve diğerleri
Baskı/Yayın Bilgisi: (2024-07-01)

T Cell-Mediated Immune Responses to AAV and AAV Vectors
Yazar:: Hildegund C. J. Ertl
Baskı/Yayın Bilgisi: (2021-04-01)

Rational Design of AAV-rh74, AAV3B, and AAV8 with Limited Liver Targeting
Yazar:: Christopher Chan, ve diğerleri
Baskı/Yayın Bilgisi: (2023-10-01)

Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models
Yazar:: Amel Saoudi, ve diğerleri
Baskı/Yayın Bilgisi: (2023-03-01)

Extensive and prolonged restoration of dystrophin expression with vivo-morpholino-mediated multiple exon skipping in dystrophic dogs.
Yazar:: Yokota, T, ve diğerleri
Baskı/Yayın Bilgisi: (2012)

Antisense Oligonucleotide-mediated Exon Skipping as a Systemic Therapeutic Approach for Recessive Dystrophic Epidermolysis Bullosa
Yazar:: Jeroen Bremer, ve diğerleri
Baskı/Yayın Bilgisi: (2016-01-01)

U1 snRNA associates with TFIIH and regulates transcriptional initiation.
Yazar:: Kwek, K, ve diğerleri
Baskı/Yayın Bilgisi: (2002)

Mechanism of U6 snRNA oligouridylation by human TUT1
Yazar:: Seisuke Yamashita, ve diğerleri
Baskı/Yayın Bilgisi: (2023-08-01)

Crystal structures of U6 snRNA-specific terminal uridylyltransferase
Yazar:: Seisuke Yamashita, ve diğerleri
Baskı/Yayın Bilgisi: (2017-06-01)

Rescu of severely affected dystrophin/utrophin deficient mice by morpholino-oligomer mediated exon skipping
Yazar:: Goyenvalle, A, ve diğerleri
Baskı/Yayın Bilgisi: (2010)

AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery
Yazar:: Sanna Koponen, ve diğerleri
Baskı/Yayın Bilgisi: (2023-07-01)