Prospects for transgenesis in the chick [PDF]
, 2004 Research to develop a useful method for genetic modification of the chick has been on-going since the first demonstrations in the mouse in the 1980s that genetic modification is an invaluable tool for the study of gene function. Manipulation of the chick
Bosselman +52 more
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Potent CRISPR-Cas9 inhibitors from Staphylococcus genomes. [PDF]
, 2020 Anti-CRISPRs (Acrs) are small proteins that inhibit the RNA-guided DNA targeting activity of CRISPR-Cas enzymes. Encoded by bacteriophage and phage-derived bacterial genes, Acrs prevent CRISPR-mediated inhibition of phage infection and can also block ...
Doudna, Jennifer A +5 more
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BMC Biotechnology, 2009 Background Lentiviral vectors are well suited for gene therapy because they can mediate long-term expression in both dividing and nondividing cells. However, lentiviral vectors seem less suitable for liver gene therapy because systemically administered ...
Hiralall Johan K +4 more
doaj +1 more source
Nucleolar Localization of HIV-1 Rev Is Required, Yet Insufficient for Production of Infectious Viral Particles. [PDF]
, 2018 Combination antiretroviral therapy fails in complete suppression of HIV-1 due to drug resistance and persistent latency. Novel therapeutic intervention requires knowledge of intracellular pathways responsible for viral replication, specifically those ...
Arizala, Jerlisa Ann C +5 more
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Effectiveness of gene delivery systems for pluripotent and differentiated cells
Molecular Therapy: Methods & Clinical Development, 2015 Human embryonic stem cells (hESC) and induced pluripotent stem cells (hiPSC) assert a great future for the cardiovascular diseases, both to study them and to explore therapies. However, a comprehensive assessment of the viral vectors used to modify these
Kleopatra Rapti +6 more
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Generation of stably expressing IRF5 spliced isoform in Jurkat cells
Progress in Microbes and Molecular Biology, 2020 Lentiviral transduction enables the generation of gain-of-function of a targeted gene in mammalian cells. Single-cell cloning through limiting dilution can establish a population of cells with homogenous transgene expression for exploring protein ...
Ashwinder Kaur, Chee-Mun Fang
doaj +1 more source
Creating New β-Globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences. [PDF]
, 2020 Hematopoietic stem cell gene therapy is a promising approach for treating disorders of the hematopoietic system. Identifying combinations of cis-regulatory elements that do not impede packaging or transduction efficiency when included in lentiviral ...
Aleshe, Bamidele +14 more
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Perinatal Gene Transfer to the Liver [PDF]
, 2011 The liver acts as a host to many functions hence raising the possibility that any one may be compromised by a single gene defect. Inherited or de novo mutations in these genes may result in relatively mild diseases or be so devastating that death within
Buckley, SM +6 more
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Intrabody Gene Therapy Ameliorates Motor, Cognitive, and Neuropathological Symptoms in Multiple Mouse Models of Huntington's Disease [PDF]
, 2009 Huntington's disease (HD) is an autosomal dominant neurodegenerative disease resulting from the expansion of a glutamine repeat in the huntingtin (Htt) protein.
Ko, Jan +2 more
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Superior lentiviral vectors designed for BSL-0 environment abolish vector mobilization. [PDF]
, 2018 Lentiviral vector mobilization following HIV-1 infection of vector-transduced cells poses biosafety risks to vector-treated patients and their communities.
Bi, Yanmin +7 more
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