Cargando…

AAV-Genome Population Sequencing of Vectors Packaging CRISPR Components Reveals Design-Influenced Heterogeneity

The gene therapy field has been galvanized by two technologies that have revolutionized treating genetic diseases: vectors based on adeno-associated viruses (AAVs), and clustered regularly interspaced short palindromic repeats (CRISPR)-Cas gene-editing tools. When combined into one platform, these s...

Descripción completa

Detalles Bibliográficos
Autores principales:	Tran, Ngoc Tam, Heiner, Cheryl, Weber, Kristina, Weiand, Michael, Wilmot, Daniella, Xie, Jun, Wang, Dan, Brown, Alexander, Manokaran, Sangeetha, Su, Qin, Zapp, Maria L., Gao, Guangping, Tai, Phillip W.L.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7397707/ https://www.ncbi.nlm.nih.gov/pubmed/32775498 http://dx.doi.org/10.1016/j.omtm.2020.07.007

Ejemplares similares

Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras
por: Tai, Phillip W.L., et al.
Publicado: (2018)

Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction
por: Muhuri, Manish, et al.
Publicado: (2021)

Efficacious, safe, and stable inhibition of corneal neovascularization by AAV-vectored anti-VEGF therapeutics
por: Su, Wenqi, et al.
Publicado: (2021)

Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?
por: Zhan, Wei, et al.
Publicado: (2021)

CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector
por: Murlidharan, Giridhar, et al.
Publicado: (2016)

CRISPR/Cas9 as an antiviral against Orthopoxviruses using an AAV vector
por: Siegrist, Cathryn M., et al.
Publicado: (2020)

Transcriptome Profiling of Neovascularized Corneas Reveals miR-204 as a Multi-target Biotherapy Deliverable by rAAVs
por: Lu, Yi, et al.
Publicado: (2018)

High levels of AAV vector integration into CRISPR-induced DNA breaks
por: Hanlon, Killian S., et al.
Publicado: (2019)

Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy
por: Yang, Yeon-Suk, et al.
Publicado: (2020)

Recombinant AAV Vectors for Enhanced Expression of Authentic IgG
por: Fuchs, Sebastian P., et al.
Publicado: (2016)

Preventing packaging of translatable P5-associated DNA contaminants in recombinant AAV vector preps
por: Brimble, Mark A., et al.
Publicado: (2022)

Quantitative analysis of genome packaging in recombinant AAV vectors by charge detection mass spectrometry
por: Barnes, Lauren F., et al.
Publicado: (2021)

CRISPR Activation Enhances In Vitro Potency of AAV Vectors Driven by Tissue-Specific Promoters
por: McDougald, Devin S., et al.
Publicado: (2019)

T Cell-Mediated Immune Responses to AAV and AAV Vectors
por: Ertl, Hildegund C. J.
Publicado: (2021)

Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing
por: Yip, Mitchell, et al.
Publicado: (2023)

Large-scale molecular epidemiological analysis of AAV in a cancer patient population
por: Qin, Wanru, et al.
Publicado: (2021)

Innate Immune Responses to AAV Vectors
por: Rogers, Geoffrey L., et al.
Publicado: (2011)

AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6
por: Denard, Jérôme, et al.
Publicado: (2018)

Rational engineering of a functional CpG-free ITR for AAV gene therapy
por: Pan, Xiufang, et al.
Publicado: (2022)

Self-delivering CRISPR RNAs for AAV Co-delivery and Genome Editing in vivo
por: Zhang, Han, et al.
Publicado: (2023)

Long-term, efficient inhibition of microRNA function in mice using rAAV vectors
por: Xie, Jun, et al.
Publicado: (2012)

Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis
por: Yang, Yeon-Suk, et al.
Publicado: (2019)

Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors
por: Wang, Qizhao, et al.
Publicado: (2016)

Vaccinia virus as a subhelper for AAV replication and packaging
por: Moore, Andrea R, et al.
Publicado: (2015)

Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo
por: Ibraheim, Raed, et al.
Publicado: (2021)

AAV Vectored Immunoprophylaxis for Filovirus Infections
por: Rghei, Amira D., et al.
Publicado: (2020)

AAV gene therapy vectors in the TMJ
por: Brouxhon, Sabine M., et al.
Publicado: (2022)

Extending AAV Packaging Cargo through Dual Co-Transduction: Efficient Protein Trans-Splicing at Low Vector Doses
por: Ferreira, Mariana V., et al.
Publicado: (2023)

Targeting the somatosensory system with AAV9 and AAV2retro viral vectors
por: Skorput, Alexander G. J., et al.
Publicado: (2022)

Viral vector platforms within the gene therapy landscape
por: Bulcha, Jote T., et al.
Publicado: (2021)

Naturally occurring singleton residues in AAV capsid impact vector performance and illustrate structural constraints
por: Vandenberghe, Luk H., et al.
Publicado: (2009)

Serotype-dependent recombinant adeno-associated vector (AAV) infection of Epstein–Barr virus-positive B-cells, towards recombinant AAV-based therapy of focal EBV + lymphoproliferative disorders
por: Ahmadi, Elham, et al.
Publicado: (2021)

Cross-Packaging and Capsid Mosaic Formation in Multiplexed AAV Libraries
por: Schmit, Pauline F., et al.
Publicado: (2019)

CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice
por: Ohmori, Tsukasa, et al.
Publicado: (2017)

Near-perfect infectivity of wild-type AAV as benchmark for infectivity of recombinant AAV vectors
por: Zeltner, Nadja, et al.
Publicado: (2010)

Successful Transduction with AAV Vectors after Selective Depletion of Anti-AAV Antibodies by Immunoadsorption
por: Orlowski, Alejandro, et al.
Publicado: (2020)

A strategy for high antibody expression with low anti-drug antibodies using AAV9 vectors
por: Davis-Gardner, Meredith E., et al.
Publicado: (2023)

AAV Vectors Vaccines Against Infectious Diseases
por: Nieto, Karen, et al.
Publicado: (2014)

AAV vector-mediated in vivo reprogramming into pluripotency
por: Senís, Elena, et al.
Publicado: (2018)

AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria
por: Richards, Daelyn Y., et al.
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_61qvg9s66gt16hk3j2e8ajbl18