Gene therapy: the first decade

Mountain, Andrew

doi:10.1016/s0167-7799(99)01416-x

Cited by 305 publications

(223 citation statements)

References 75 publications

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“…The ability to correct these genetic defects would provide an advancement in the treatment of diseases caused by genetic malfunctions (Friedmann and Roblin 1972). Gene therapy is a promising strategy of treating diseases at a genetic level by introducing genes to a target cell in order to correct, restore, modify or enhance cellular activities (Mountain 2000;Nathwani, Benjamin et al 2004). Gene therapy employs nucleic acid to prevent or treat acquired diseases such as cancer (McNeish, Bell et al 2004), neurological diseases (Ribotta 2001), cardiovascular diseases (Katz, Swain et al 2010) or inherited diseases such as muscular dystrophy (Inui, Okada et al 1996) or cystic fibrosis (Griesenbach, Geddes et al 2006).…”

Section: Introductionmentioning

confidence: 99%

Evaluation of Cell-Penetrating Peptide/Adenovirus Particles for Transduction of CAR-Negative Cells

Nigatu

Vupputuri

Flynn

et al. 2013

Journal of Pharmaceutical Sciences

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Linear low‐density polyethylene (LLDPE), based on butene‐1 or hexene‐1, was irradiated with γ‐rays under vacuum or in the presence of air. The study focused on the influence of the dose rate and the γ‐dose on the thermal properties of LLDPE. Differential scanning calorimetry, thermogravimetric analysis (TGA), and TGA/FTIR techniques were used to address the thermal behavior as a result of γ‐irradiation. During this irradiation, competition between crosslinking and scission reactions, subsequent to oxidation reactions, occurred in the polymeric material, which strongly depends on the experimental conditions. A decrease of the crystallinity for γ‐irradiated samples was observed in particular for samples irradiated under vacuum. This observation may be explained by increased hindrance of segment mobility due to crosslinking reactions that prevent crystal growth. TGA investigations revealed an enhancement of the thermal stability for samples irradiated under vacuum but not for those irradiated in air. © 2006 Wiley Periodicals, Inc. J Appl Polym Sci 100: 2790–2795, 2006

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Section: Introductionmentioning

confidence: 99%

Evaluation of Cell-Penetrating Peptide/Adenovirus Particles for Transduction of CAR-Negative Cells

Nigatu

Vupputuri

Flynn

et al. 2013

Journal of Pharmaceutical Sciences

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“…[1][2][3][4] Lentiviral vectors have the unique feature of being able to transduce nondividing cells, making it particularly attractive for certain gene therapy applications. [5][6][7] Sometimes, to achieve a desirable therapeutic effect, the viral vectors must be capable of precisely delivering a gene of interest to specific cells without influencing non-target cells.…”

Section: Introductionmentioning

confidence: 99%

Visualization of targeted transduction by engineered lentiviral vectors

Joo

Wang

2008

Gene Ther

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“…The generation of high titer and quality retroviral vector (RV) preparations are currently critical issues in gene therapy clinical applications [1,2].…”

Section: Introductionmentioning

confidence: 99%

Removal of envelope protein‐free retroviral vectors by anion‐exchange chromatography to improve product quality

Rodrigues

Alves

Lopes

et al. 2008

J of Separation Science

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Original PaperRemoval of envelope protein-free retroviral vectors by anion-exchange chromatography to improve product qualityWe have investigated the role of the retroviral lipid bilayer and envelope proteins in the adsorption of retroviral vectors (RVs) to a Fractogel TM DEAE matrix. Intact RVs and their degradation components (envelope protein-free vectors and solubilized vector components) were adsorbed to this matrix and eluted using a linear gradient. Envelope protein-free RVs (Env -) and soluble envelope proteins (gp70) eluted in a significantly lower range of conductivities than intact RVs (Env + ) (13.7 -30 mS/cm for Env -and gp70 proteins vs. 47 -80 mS/cm for Env + ). The zeta (f)-potential of Env + and Env -vectors was evaluated showing that envelope proteins define the pI of the viral particles (pI (Env + ) a 2 versus 3 a pI (Env -) a 4) and that Env + and Env -vectors have similar f-potentials within pH 5 and 8. The results presented herein indicate that the adsorption of retroviral particles occurs through multi-point interaction of the envelope proteins with the cationic groups on the chromatographic matrix. The strength of this adsorption is thus dependent on the amount of envelope protein present in the viral lipid bilayer. In conclusion, AEXc enables the separation of gp70 proteins as well as envelope protein-free vectors constituting a significant improvement to the quality of retroviral preparations for gene therapy applications.

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Gene therapy: the first decade

Cited by 305 publications

References 75 publications

Evaluation of Cell-Penetrating Peptide/Adenovirus Particles for Transduction of CAR-Negative Cells

Evaluation of Cell-Penetrating Peptide/Adenovirus Particles for Transduction of CAR-Negative Cells

Visualization of targeted transduction by engineered lentiviral vectors

Removal of envelope protein‐free retroviral vectors by anion‐exchange chromatography to improve product quality

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