Episomal Gene Delivery

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Episomal vectors for gene expression in mammalian cells ...

    https://febs.onlinelibrary.wiley.com/doi/full/10.1046/j.1432-1327.2000.01645.x
    Dec 25, 2001 · Gene transfer by nonviral vector‐mediated systems has been shown to be a safe and simple but relatively inefficient method for gene delivery in vivo[]. Many of the systems currently available utilize viral vectors derived from retroviruses, lentiviruses, adenoviruses, etc. [[21, 171]]. We will focus on the utility of nonintegrating viruses ...Author: Kathleen Van Craenenbroeck, Peter Vanhoenacker, Guy Haegeman

Viral Delivery Systems for CRISPR

    https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6356701/
    Jan 04, 2019 · As a delivery vector for CRISPR/Cas9-based gene editing, this limits potential off-target effects, which are unwanted mutations at sites other than the designated target site. Due to high transduction efficiency and minimal human symptoms, numerous efforts have been made to optimize the AdV as a gene delivery vector.Author: Christine L. Xu, Merry Z. C. Ruan, Vinit B. Mahajan, Stephen H. Tsang

Episomal vectors for gene therapy.

    https://www.ncbi.nlm.nih.gov/pubmed/18537590
    The main goal of gene therapy is to treat loss-of-function genetic disorders by delivering correcting therapeutic DNA sequences into the nucleus of a cell, allowing its long-term expression at physiologically relevant levels. Manifold different vector systems for the therapeutic gene delivery have been described over the recent years.Author: Savio L. C. Woo, Peter W. Nordloh, Arne Stenlund

Comparison of Gene Delivery to the Kidney by Adenovirus ...

    https://www.liebertpub.com/doi/10.1089/hum.2019.127
    Dec 16, 2019 · There are many kidney diseases that might be addressed by gene therapy. However, gene delivery to kidney cells is inefficient. This is due, in part, to the fact that the kidney excludes molecules above 50 kDa and that most gene delivery vectors are megaDaltons in mass.Author: Jeffrey D. Rubin, Tien V. Nguyen, Kari L. Allen, Katayoun Ayasoufi, Michael A. Barry

GENE DELIVERY & EXPRESSION - System Biosciences

    https://www.systembio.com/wp-content/uploads/SBI_Brochure_Gene_Delivery_and_Expression.pdf
    for gene delivery and expression. From our popular high-titer lentivirus production tools to the latest in AAV-based gene delivery systems and a range of reliable integrating and non-integrating gene expression vectors, SBI delivers high-quality, cutting-edge products and services for mammalian gene delivery and expression.

142 Development of an S/MAR based episomal vector for the ...

    https://www.sciencedirect.com/science/article/pii/S1569199315303192
    S94 142 Development of an S/MAR based episomal vector for the CFTR gene delivery 5. New Therapies Posters 143 Lumacaftor/ivacaftor combination therapy in CF patients homozygous for F508del-CFTR with severe lung dysfunction J.S. Elborn1 , B. Ramsey2 , M.P. Boyle3 , C. Wainwright4 , M. Konstan5 , X. Huang6 , G. Marigowda6 , D. Waltz6 , on behalf of the VX-809 TRAFFIC & TRANSPORT Study …Author: D. De Rocco, B. Pompili, N. Cifani, E.G. DiDomenico, P. Del Porto, F. Ascenzioni

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