Comparative genomic integration profiling of Sleeping Beauty transposons mobilized with high efficacy from integrase-defective lentiviral vectors in primary human …

B Moldt, C Miskey, NH Staunstrup, A Gogol-Döring… - Molecular Therapy, 2011 - cell.com
It has been previously shown that integrase-defective HIV-1-based gene vectors can serve,
with moderate efficiency, as substrate for DNA transposition by a transiently expressed …

Genome-wide target profiling of piggyBac and Tol2in HEK 293: pros and cons for gene discovery and gene therapy

YJJ Meir, MT Weirauch, HS Yang, PC Chung, RK Yu… - BMC …, 2011 - Springer
Background DNA transposons have emerged as indispensible tools for manipulating
vertebrate genomes with applications ranging from insertional mutagenesis and …

Enhanced guide-RNA design and targeting analysis for precise CRISPR genome editing of single and consortia of industrially relevant and non-model organisms

BJ Mendoza, CT Trinh - Bioinformatics, 2018 - academic.oup.com
Motivation Genetic diversity of non-model organisms offers a repertoire of unique phenotypic
features for exploration and cultivation for synthetic biology and metabolic engineering …

Preclinical efficacy and safety of CD19CAR cytokine-induced killer cells transfected with sleeping beauty transposon for the treatment of acute lymphoblastic leukemia

CF Magnani, C Mezzanotte, C Cappuzzello… - Human gene …, 2018 - liebertpub.com
Infusion of patient-derived CD19-specific chimeric antigen receptor (CAR) T cells
engineered by viral vectors achieved complete remission and durable response in relapsed …

Bats with hATs: Evidence for Recent DNA Transposon Activity in Genus Myotis

DA Ray, HJT Pagan, ML Thompson… - Molecular biology and …, 2007 - academic.oup.com
Transposable elements make up a significant fraction of many eukaryotic genomes.
Although both classes of transposable elements, the DNA transposons and the …

Sleeping beauty transposition from nonintegrating lentivirus

CA Vink, HB Gaspar, R Gabriel, M Schmidt, RS McIvor… - Molecular therapy, 2009 - cell.com
Lentiviral vectors enter cells with high efficiency and deliver stable transduction through
integration into host chromosomes, but their preference for integration within actively …

Excision of Sleeping Beauty transposons: parameters and applications to gene therapy

G Liu, EL Aronovich, Z Cui, CB Whitley… - The Journal of Gene …, 2004 - Wiley Online Library
A major problem in gene therapy is the determination of the rates at which gene transfer has
occurred. Our work has focused on applications of the Sleeping Beauty (SB) transposon …

International review of cell and molecular biology

KW Jeon - 2013 - books.google.com
International Review of Cell and Molecular Biology presents current advances and
comprehensive reviews in cell biology--both plant and animal. Articles address structure and …

Potentiation of ABCA3 lipid transport function by ivacaftor and genistein

S Kinting, Y Li, M Forstner, F Delhommel… - Journal of cellular …, 2019 - Wiley Online Library
ABCA3 is a phospholipid transporter implicated in pulmonary surfactant homoeostasis and
localized at the limiting membrane of lamellar bodies, the storage compartment for surfactant …

ABCA3 missense mutations causing surfactant dysfunction disorders have distinct cellular phenotypes

U Schindlbeck, T Wittmann, S Höppner… - Human …, 2018 - Wiley Online Library
Mutations in the ATP‐binding cassette subfamily A member 3 (ABCA3) gene are the most
common monogenetic cause of surfactant dysfunction disorders in newborns and interstitial …