Progress and prospects: gene therapy clinical trials (part 1)

Gene Therapy (Impact Factor: 4.2). 11/2007; 14(20):1439-47. DOI: 10.1038/
Source: PubMed

ABSTRACT Over the last two decades gene therapy has moved from preclinical to clinical studies for many diseases ranging from single gene disorders such as cystic fibrosis and Duchenne muscular dystrophy, to more complex diseases such as cancer and cardiovascular disorders. Gene therapy for severe combined immunodeficiency (SCID) is the most significant success story to date, but progress in many other areas has been significant. We asked 20 leaders in the field succinctly to summarize and comment on clinical gene therapy research in their respective areas of expertise and these are published in two parts in the Progress and Prospect series.

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Available from: Seng H. Cheng, Aug 26, 2015
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    • "Whole cells may also be the target product for example in stem cell therapy [Weber et al., 2007b]. Insect cells, which are for example used for the production of recombinant proteins, viruses or viral components [Nehring et al., 2006] are currently gaining an increased importance in the field of regenerative medicine [Negrete and Kotin, 2008, Stanbridge et al., 2003], especially in gene therapy [Alexander et al., 2007, Edelstein et al., 2007, Stanbridge, et al., 2003]. Cells may be grown in suspension, on surfaces, or even in threedimensional solid matrices. "
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    ABSTRACT: In the biopharmaceutical industry, mammalian and insect cells as well as plant cell cultures are gaining worldwide importance to produce biopharmaceuticals and as products themselves, for example in stem cell therapy. These highly sophisticated cell-based production processes need to be monitored and controlled to guarantee product quality and to satisfy GMP requirements. With the process analytical technology (PAT) initiative, requirements regarding process monitoring and control have changed and real-time in-line monitoring tools are now recommended. Dielectric spectroscopy (DS) can serve as a tool to satisfy some PAT requirements. DS has been used in the medical field for quite some time and it may allow real-time process monitoring of biological cell culture parameters. DS has the potential to enable process optimization, automation, cost reduction, and a more consistent product quality. Dielectric spectroscopy is reviewed here as a tool to monitor biochemical processes. Commercially available dielectric sensing systems are discussed. The potential of this technology is demonstrated through examples of current and potential future applications in research and industry for mammalian and insect cell culture.
    Biotechnology advances 03/2011; 29(4):391-401. DOI:10.1016/j.biotechadv.2011.03.002 · 8.91 Impact Factor
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    • "Recombinant retroviruses such as murine leukemia viruses (MLV) are promising biotechnological tools and have been investigated in various clinical trials [1] [2] [3] [4]. Process strategies and downstream processing of pharmaceutical vaccines and vectors expressing recombinant proteins have been established, though, due to increased degradation rates of retroviral particles, vector concentration does not reach the appropriate level for successful application in gene therapy [1–2, 5–7]. "
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    ABSTRACT: Pseudotype vectors are promising for gene transfer in many gene therapy approaches, however low vector con-centration in batch cultures and high temperature-dependent decay limit sufficiently large-scale production. The ability of commercial ceramic asymmetric ultrafiltration membranes with a cut-off of 20 kDa to purify retroviral pseudotype vectors derived from the murine leukemia virus (MLV) carrying the HIV-1 envelope protein MLV (HIV-1) was studied. Experimental study was carried out in order to analyse the impact of inline tangential flow filtration (TFF) combined with different harvest temperatures in continuous production mode compared to batch filtration. Retro-viral pseudotype vectors were produced using a 200 ml fixed bed reactor for high cell density cultivation on macro porous carriers up to 400 hours. By tangential flow ultrafiltration combined with cooling down the harvest super-natant to 4°C, the vector concentration was increased 12-fold with an average recovery of 78.7% of the initial infective capacity.
    Desalination 09/2009; 246(246):241-247. DOI:10.1016/j.desal.2009.02.027 · 3.96 Impact Factor
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    • "Gene therapy is also highly desired for the treatment of neurologic and other chronic disease. Clinical trials have been implemented and/or completed for the treatment of HIV/AIDS, arthritis, angina pectoris, solid tumors, Parkinson's disease, Huntington's disease, Alzheimer's disease, Batten disease , Canavan disease, and familial hypercholesterolemia (Aiuti et al., 2007; Alexander et al., 2007; NIH, 2008a,b). Despite the many hurdles, most clinical trials are progressing steadily, with treatments for angina pectoris (Henry et al., 2007), prostate cancer (Freytag et al., 2007), non-small-cell lung cancer, and head and neck cancer now entering phase III clinical trials (NIH, 2008b). "
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    ABSTRACT: Gene therapy is defined as the treatment of disease by transfer of genetic material into cells. This review will explore methods available for gene transfer as well as current and potential applications for craniofacial regeneration, with emphasis on future development and design. Though non-viral gene delivery methods are limited by low gene transfer efficiency, they benefit from relative safety, low immunogenicity, ease of manufacture, and lack of DNA insert size limitation. In contrast, viral vectors are nature's gene delivery machines that can be optimized to allow for tissue-specific targeting, site-specific chromosomal integration, and efficient long-term infection of dividing and non-dividing cells. In contrast to traditional replacement gene therapy, craniofacial regeneration seeks to use genetic vectors as supplemental building blocks for tissue growth and repair. Synergistic combination of viral gene therapy with craniofacial tissue engineering will significantly enhance our ability to repair and replace tissues in vivo.
    Journal of dental research 08/2009; 88(7):585-96. DOI:10.1177/0022034509337480 · 4.14 Impact Factor
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