Pseudotyped Lentivirus Vectors Derived from Simian Immunodeficiency Virus SIVagm with Envelope Glycoproteins from Paramyxovirus

Kobayashi, Masanori; Iida, Akihiro; Ueda, Yasuji; Hasegawa, Mamoru

doi:10.1128/jvi.77.4.2607-2614.2003

Cited by 70 publications

(70 citation statements)

References 30 publications

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“…The envelope proteins F (fusion) and HN (hemagglutinin-neuraminidase) are the key factors in determining the high-transfection efficiency to airways. We have, therefore, recently pseudotyped a simian lentiviral vector with the SeV F and HN proteins (F/HN-SIV) 23 and have shown that the virus efficiently transfects mouse airway epithelium in vivo, as well as human air-liquid interface cultures. 24 Moreover, F/HN-SIV-mediated gene expression persists for more than 17 months after a single administration and repeated administration (3 doses) is feasible.…”

Section: Discussionmentioning

confidence: 99%

Validation of recombinant Sendai virus in a non-natural host model

et al. 2010

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We have previously shown that recombinant Sendai virus (SeV) vector, derived from murine parainfluenza virus, is one of the most efficient vectors for airway gene transfer. We have also shown that SeV-mediated transfection on second administration, although reduced by 60% when compared with levels achieved after a single dose, is still high because of the efficient transfection achieved by SeV vector in murine airways. Here, we show that these levels further decrease on subsequent doses. In addition, we validated SeV vector repeat administration in a non-natural host model, the sheep. As part of these studies we first assessed viral stability in a Pari LC Plus nebuliser, a polyethylene catheter (PEC) and the Trudell AeroProbe. We also compared the distribution of gene expression after PEC and Trudell AeroProbe administration and quantified virus shedding after sheep transduction. In addition, we show that bronchial brushings and biopsies, collected in anaesthetized sheep, can be used to assess SeV-mediated gene expression over time. Similar to mice, gene expression in sheep was transient and had returned to baseline values by day 14. In conclusion, the SeV vector should be strongly considered for lung-related applications requiring a single administration of the vector even though it might not be suitable for diseases requiring repeat administration.

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Section: Discussionmentioning

confidence: 99%

Validation of recombinant Sendai virus in a non-natural host model

et al. 2010

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“…A lentiviral pseudotype based on the envelope proteins of the lung-tropic Sendai virus (Figure 3(b)) has shown promising results in pre-clinical models and is poised for clinical trial [36,37]. A novel lentiviral pseudotype, derived by error-prone PCR-mediated directed evolution (Figure 2(a)) of the basic GP64 pseudotype, improved expression in human primary airway epithelial cells [38].…”

Section: A Brief History Of In-vivo Gene Therapymentioning

confidence: 99%

Lessons learned from lung and liver in-vivo gene therapy: implications for the future

Haasteren

Hyde

Gill

2018

Expert Opinion on Biological Therapy

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Introduction: Ex-vivo gene therapy has had significant clinical impact over the last couple of years and in-vivo gene therapy products are being approved for clinical use. Gene therapy and gene editing approaches have huge potential to treat genetic disease and chronic illness. Areas covered: This article provides a review of in-vivo approaches for gene therapy in the lung and liver, exploiting non-viral and viral vectors with varying serotypes and pseudotypes to target-specific cells. Antibody responses inhibiting viral vectors continue to constrain effective repeat administration. Lessons learned from ex-vivo gene therapy and genome editing are also discussed. Expert opinion: The fields of lung and liver in-vivo gene therapy are thriving and a comparison highlights obstacles and opportunities for both. Overcoming immunological issues associated with repeated administration of viral vectors remains a key challenge. The addition of targeted small molecules in combination with viral vectors may offer one solution. A substantial bottleneck to the widespread adoption of in-vivo gene therapy is how to ensure sufficient capacity for clinical-grade vector production. In the future, the exploitation of gene editing approaches for in-vivo disease treatment may facilitate the resurgence of non-viral gene transfer approaches, which tend to be eclipsed by more efficient viral vectors.

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“…14,18 Recent interest has converged on pseudotyping; be it of adeno-associated virus (AAV)-2 genomes with novel AAV serotypes 21,36 or of integrating lentiviral vectors with viral envelopes from lung pathogens. 28,29,[31][32][33]37 The Ebola virus envelope glycoprotein has been successfully used to pseudotype lentivirus and has achieved efficient transduction of the murine lung epithelium and human explants 33,34 although generation of consistently high viral titres has been problematic. Recently the baculovirus gp64 envelope has been shown to produce significant expression in the nasal epithelia of adult mice up to 1 year after application.…”

Section: Discussionmentioning

confidence: 99%

“…[24][25][26][27] However, airway epithelial gene transfer efficacy with this vector system has previously been restricted by a paucity of pseudotypes, which can be produced at high titres and which can infect lung epithelia from the apical surface. In recent years, apical transduction by lentivirus has been achieved both in vitro and in vivo using a number of viral envelopes from diverse origins, including filovirus, 28,29 baculovirus, 30 influenza 31 and parainfluenza 32 viruses. For example, the Ebola virus envelope glycoprotein has been used successfully to achieve efficient transduction of the murine lung epithelium and human explants 33,34 although generation of consistently high viral titres has been problematic.…”

Section: Introductionmentioning

confidence: 99%