Cargando…

Molecular characterization of precise in vivo targeted gene integration in human cells using AAVHSC15

Targeted gene integration via precise homologous recombination (HR)-based gene editing has the potential to correct genetic diseases. AAV (adeno-associated virus) can mediate nuclease-free gene integration at a disease-causing locus. Therapeutic application of AAV gene integration requires quantitat...

Descripción completa

Detalles Bibliográficos
Autores principales:	Chen, Huei-Mei, Resendes, Rachel, Ghodssi, Azita, Sookiasian, Danielle, Tian, Michael, Dollive, Serena, Adamson-Small, Laura, Avila, Nancy, Tazearslan, Cagdas, Thompson, John F., Ellsworth, Jeff L., Francone, Omar, Seymour, Albert, Wright, Jason B.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Public Library of Science 2020
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7250422/ https://www.ncbi.nlm.nih.gov/pubmed/32453743 http://dx.doi.org/10.1371/journal.pone.0233373

Ejemplares similares

Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH
por: Ahmed, Seemin S., et al.
Publicado: (2020)

Natural variations in AAVHSC16 significantly reduce liver tropism and maintain broad distribution to periphery and CNS
por: Smith, Laura J., et al.
Publicado: (2022)

Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates
por: Ellsworth, Jeff L., et al.
Publicado: (2019)

Identification and functional validation of an enhancer variant in the 9p21.3 locus associated with glaucoma risk and elevated expression of p16(INK4a)
por: Zhu, Yizhou, et al.
Publicado: (2023)

Identification and functional validation of an enhancer variant in the 9p21.3 locus associated with glaucoma risk and elevated expression of p16(INK4a)
por: Zhu, Yizhou, et al.
Publicado: (2023)

Age- and Tissue-Specific Expression of Senescence Biomarkers in Mice
por: Hudgins, Adam D., et al.
Publicado: (2018)

Rec-8 dimorphism affects longevity, stress resistance and X-chromosome nondisjunction in C. elegans, and replicative lifespan in S. cerevisiae
por: Ayyadevara, Srinivas, et al.
Publicado: (2014)

SOX2 is required for inner ear neurogenesis
por: Steevens, Aleta R., et al.
Publicado: (2017)

5-Aza-2′-deoxycytidine-induced genome rearrangements are mediated by DNMT1
por: Maslov, Alexander Y., et al.
Publicado: (2012)

Modulation of lipid biosynthesis contributes to stress resistance and longevity of C. elegans mutants
por: Shmookler Reis, Robert J., et al.
Publicado: (2011)

The current landscape of imaging recommendations in cardiovascular clinical guidelines: toward an imaging-guided precision medicine
por: Esposito, Antonio, et al.
Publicado: (2020)

Archaea and Fungi of the Human Gut Microbiome: Correlations with Diet and Bacterial Residents
por: Hoffmann, Christian, et al.
Publicado: (2013)

Pena de muerte : la controversia /
por: Resendes Macías, Víctor Hugo
Publicado: (2001)

Second International Lygus Symposium: Asilomar Conference Grounds, Pacific Grove, California, April 15-19 2007
por: Goodell, P. B., et al.
Publicado: (2008)

Positive Feedback between Transcriptional and Kinase Suppression in Nematodes with Extraordinary Longevity and Stress Resistance
por: Tazearslan, Çagdaþ, et al.
Publicado: (2009)

Concussion reporting behaviours of community college student-athletes and limits of transferring concussion knowledge during the stress of competition
por: Chinn, Nancy Resendes, et al.
Publicado: (2016)

Exposure to surrounding greenness and well-being in Portugal, a cross sectional study
por: Resendes, D S, et al.
Publicado: (2023)

Precision Medicine in Radiomics and Radiogenomics
por: Monti, Serena
Publicado: (2022)

15th International Conference of the European Society for Precision Engineering and Nanotechnology
por: Leach, R
Publicado: (2015)

Results of 15 Years of Precision Feeding of Hyper Prolific Gestating Sows
por: Quiniou, Nathalie
Publicado: (2021)

Fungi of the Murine Gut: Episodic Variation and Proliferation during Antibiotic Treatment
por: Dollive, Serena, et al.
Publicado: (2013)

Precision medicine for all? Challenges and opportunities for a precision medicine approach to critical illness
por: Seymour, Christopher W., et al.
Publicado: (2017)

Precise wide range heatmeters for 1.5 K up to 80 K
por: Ferlin, G, et al.
Publicado: (1998)

Estimating short-term and long-term survival in rectal cancer patients using cure model
por: Beiranvand, Behrouz, et al.
Publicado: (2022)

Adsorption Kinetic Model Predicts and Improves Reliability of Electrochemical Serotonin Detection
por: Chapin, Ashley Augustiny, et al.
Publicado: (2023)

In Young Adults with COVID-19, Obesity Is Associated with Adverse Outcomes
por: Steinberg, Eric, et al.
Publicado: (2020)

5th International Conference on the Physics of Dusty Plasmas
por: Mendonça, José Tito, et al.
Publicado: (2008)

Reduced parenting stress following a prevention program decreases internalizing and externalizing symptoms in the offspring of parents with bipolar disorder
por: Resendes, Tiffany, et al.
Publicado: (2023)

News on Task 15.2: Improvements of test beam infrastructure for high precision tracking
por: Dreyling-Eschweiler, Jan
Publicado: (2018)

Precise Expression of Afmed15 Is Crucial for Asexual Development, Virulence, and Survival of Aspergillus fumigatus
por: Guan, Luyu, et al.
Publicado: (2020)

Hepatic inflammation resulting from localized, subcutaneous expression of Parvovirus VP1u capsid protein: Importance and implication
por: Adamson-Small, Laura A, et al.
Publicado: (2016)

Adobe Audition 1.5 for Windows
por: Partyka, Jeff
Publicado: (2005)

Statistical Significance of Precisely Repeated Intracellular Synaptic Patterns
por: Ikegaya, Yuji, et al.
Publicado: (2008)

The utility of 1.5 tesla MR-guided adaptive stereotactic body radiotherapy for recurrent ovarian tumor – Case reports and review of the literature
por: Yavas, Guler, et al.
Publicado: (2022)

Feather mites (Acari, Astigmata) from Azorean passerines (Aves, Passeriformes): lower species richness compared to European mainland
por: Rodrigues, Pedro, et al.
Publicado: (2015)

Improved cell-specificity of adeno-associated viral vectors for medullary thyroid carcinoma using calcitonin gene regulatory elements
por: Levy, Hazel C., et al.
Publicado: (2020)

Vitamin and mineral supplements: a survey of knowledge, attitudes and behaviors among Southern Utah University students, faculty and staff
por: Ellsworth, Kamber, et al.
Publicado: (2008)

Cardiac angiosarcoma treated with 1.5 Tesla MR-guided adaptive stereotactic body radiotherapy – Case report and review of the literature
por: Noyan, Asli, et al.
Publicado: (2022)

Role of Cardiac Magnetic Resonance in the Evaluation of Dilated Cardiomyopathy: Diagnostic Contribution and Prognostic Significance
por: Francone, Marco
Publicado: (2014)

Core Audio
por: Avila, Kevin, et al.
Publicado: (2011)

Cannot write session to /tmp/vufind_sessions/sess_tuudo6hf1a9vvnqfabj1bt7988