Cargando…

Characterization of the humanized FRG mouse model and development of an AAV-LK03 variant with improved liver lobular biodistribution

Recent clinical successes have intensified interest in using adeno-associated virus (AAV) vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical physiological functions and involvement in a wide range of genetic diseases. In the present study, we first investig...

Descripción completa

Detalles Bibliográficos
Autores principales:	Cabanes-Creus, Marti, Navarro, Renina Gale, Liao, Sophia H.Y., Scott, Suzanne, Carlessi, Rodrigo, Roca-Pinilla, Ramon, Knight, Maddison, Baltazar, Grober, Zhu, Erhua, Jones, Matthew, Denisenko, Elena, Forrest, Alistair R.R., Alexander, Ian E., Tirnitz-Parker, Janina E.E., Lisowski, Leszek
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9860073/ https://www.ncbi.nlm.nih.gov/pubmed/36700121 http://dx.doi.org/10.1016/j.omtm.2022.12.014

Ejemplares similares

Single amino acid insertion allows functional transduction of murine hepatocytes with human liver tropic AAV capsids
por: Cabanes-Creus, Marti, et al.
Publicado: (2021)

Novel human liver-tropic AAV variants define transferable domains that markedly enhance the human tropism of AAV7 and AAV8
por: Cabanes-Creus, Marti, et al.
Publicado: (2021)

AAV-p40 Bioengineering Platform for Variant Selection Based on Transgene Expression
por: Westhaus, Adrian, et al.
Publicado: (2022)

Attenuation of Heparan Sulfate Proteoglycan Binding Enhances In Vivo Transduction of Human Primary Hepatocytes with AAV2
por: Cabanes-Creus, Marti, et al.
Publicado: (2020)

High-Throughput In Vitro, Ex Vivo, and In Vivo Screen of Adeno-Associated Virus Vectors Based on Physical and Functional Transduction
por: Westhaus, Adrian, et al.
Publicado: (2020)

Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort
por: Perocheau, Dany P., et al.
Publicado: (2019)

Regional risk factors for stomach cancer in the FRG.
por: Boeing, H, et al.
Publicado: (1991)

L.K. Batteries
Publicado: (1893)

The transition from HK to LK phenotype in the red cells of newborn genetically LK lambs
Publicado: (1982)

Transcriptomic Insights into Functions of LkABCG36 and LkABCG40 in Nicotiana tabacum
por: Sun, Nan, et al.
Publicado: (2023)

Decreased Proliferation Kinetics of Mouse Myoblasts Overexpressing FRG1
por: Chen, Steven C., et al.
Publicado: (2011)

LkARF7 and LkARF19 overexpression promote adventitious root formation in a heterologous poplar model by positively regulating LkBBM1
por: Tao, Gui-Yun, et al.
Publicado: (2023)

Reduced expression of FRG1 facilitates breast cancer progression via GM-CSF/MEK-ERK axis by abating FRG1 mediated transcriptional repression of GM-CSF
por: Mukherjee, Bratati, et al.
Publicado: (2022)

Characterization and biodistribution of under-employed gene therapy vector AAV7
por: Yost, Samantha A., et al.
Publicado: (2023)

Absence of Superconductivity in LK-99 at Ambient Conditions
por: Kumar, Kapil, et al.
Publicado: (2023)

DUX4 promotes transcription of FRG2 by directly activating its promoter in facioscapulohumeral muscular dystrophy
por: Thijssen, Peter E, et al.
Publicado: (2014)

Efficient engraftment and viral transduction of human hepatocytes in an FRG rat liver humanization model
por: Carbonaro, Marisa, et al.
Publicado: (2022)

LK or IGF1R: When selectivity hurts
por: Puzanov, Igor, et al.
Publicado: (2015)

Role of FRG1 in predicting the overall survivability in cancers using multivariate based optimal model
por: Khan, Rehan, et al.
Publicado: (2021)

Systemic AAV6-synapsin-GFP administration results in lower liver biodistribution, compared to AAV1&2 and AAV9, with neuronal expression following ultrasound-mediated brain delivery
por: Weber-Adrian, Danielle, et al.
Publicado: (2021)

FHL1 Reduces Dystrophy in Transgenic Mice Overexpressing FSHD Muscular Dystrophy Region Gene 1 (FRG1)
por: Feeney, Sandra J., et al.
Publicado: (2015)

Interaction of HK and LK Goat Red Blood Cells with Ouabain
por: Sachs, John R., et al.
Publicado: (1974)

Cytodifferentiation and Membrane Transport Properties in LK Sheep Red Cells
por: Lee, Ping, et al.
Publicado: (1966)

Transgenic Drosophila for Investigating DUX4 and FRG1, Two Genes Associated with Facioscapulohumeral Muscular Dystrophy (FSHD)
por: Jones, Takako I., et al.
Publicado: (2016)

Reduced FRG1 expression promotes prostate cancer progression and affects prostate cancer cell migration and invasion
por: Tiwari, Ankit, et al.
Publicado: (2019)

Reduced FRG1 expression promotes angiogenesis via activation of the FGF2‐mediated ERK/AKT pathway
por: Mukherjee, Bratati, et al.
Publicado: (2023)

Laccase produced by a thermotolerant strain of Trametes trogii LK13
por: Yan, Jinping, et al.
Publicado: (2015)

Location and Conformation of the LKα14 Peptide in Water/Ethanol Mixtures
por: Maltseva, Daria, et al.
Publicado: (2020)

The Formation and Properties of Thin Lipid Membranes from HK and LK Sheep Red Cell Lipids
por: Andreoli, Thomas E., et al.
Publicado: (1967)

Biodistribution of adeno-associated virus serotype 9 (AAV9) vector after intrathecal and intravenous delivery in mouse
por: Schuster, Daniel J., et al.
Publicado: (2014)

Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease
por: Wang, Gensheng, et al.
Publicado: (2014)

LK sheep reticulocytosis: effect of anti-L on K influx and in vitro maturation
Publicado: (1980)

The Effect of Valinomycin on Potassium and Sodium Permeability of HK and LK Sheep Red Cells
por: Tosteson, Daniel C., et al.
Publicado: (1967)

The Newly Isolated Endophytic Fungus Paraconiothyrium sp. LK1 Produces Ascotoxin
por: Khan, Abdul Latif, et al.
Publicado: (2012)

Assessment of Pre-Clinical Liver Models Based on Their Ability to Predict the Liver-Tropism of Adeno-Associated Virus Vectors
por: Westhaus, Adrian, et al.
Publicado: (2023)

Corrigendum to “Assessment of toxicity and biodistribution of recombinant AAV8 vector–mediated immunomodulatory gene therapy in mice with Pompe disease”
por: Wang, Gensheng, et al.
Publicado: (2015)

Preclinical biodistribution, tropism, and efficacy of oligotropic AAV/Olig001 in a mouse model of congenital white matter disease
por: Francis, Jeremy S., et al.
Publicado: (2021)

Activation of Cdc42 by trans interactions of the cell adhesion molecules nectins through c-Src and Cdc42-GEF FRG
por: Fukuhara, Tatsuro, et al.
Publicado: (2004)

Rbfox1 Downregulation and Altered Calpain 3 Splicing by FRG1 in a Mouse Model of Facioscapulohumeral Muscular Dystrophy (FSHD)
por: Pistoni, Mariaelena, et al.
Publicado: (2013)

Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles
por: Palazzi, Xavier, et al.
Publicado: (2022)

Cannot write session to /tmp/vufind_sessions/sess_75se1saua7guush8elovif5muq