Ultrasound-assisted nonviral gene transfer of AQP1 to the irradiated minipig parotid gland restores fluid secretion

Wang, Z; Zourelias, Lee; Wu, Changgong; Edwards, Paul C.; Trombetta, Mark; Passineau, Michael J.

doi:10.1038/gt.2015.36

Cited by 43 publications

(60 citation statements)

References 37 publications

(43 reference statements)

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“…Thus, using the same expression cassette and equimolar amounts of gene, the authors found that UAGT with minicircles eliminated >95% of the protein changes associated with plasmid vectors. As noted above, Passineau and colleagues have extended these studies to examine delivery of the AQP1 cDNA to irradiated porcine parotid glands with considerable success [27], essentially replicating the results of Shan et al [37] without requiring the need to employ an Ad5 vector. These results offer considerable promise for future uses for sonoporation, microbubbles and minicircles in all clinical salivary gene transfer applications.…”

Section: Methodological Advancesmentioning

confidence: 87%

“…Also of particular note, very recent studies by Wang et al [27], used ultrasound-assisted gene transfer (UAGT) to deliver both the human and porcine AQP1 cDNAs to irradiated miniature pig parotid glands. They showed that this non-viral gene transfer approach can lead to increased parotid saliva flow from the radiation damaged glands, with levels approximating those achieved with Ad5-mediated hAQP1 cDNA transfer.…”

Section: Glandular Disordersmentioning

confidence: 99%

“…Multiple non-viral methods including cationic liposomes [86], ultrasound assisted microbubble destruction [27], pH-dependent nanoparticles [35]…”

Section: Tablementioning

confidence: 99%

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Advances in salivary gland gene therapy – oral and systemic implications

Baum¹,

Alevizos²,

Chiorini³

et al. 2015

Expert Opinion on Biological Therapy

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Introduction Much research demonstrates the feasibility and efficacy of gene transfer to salivary glands. Recently, the first clinical trial targeting a salivary gland was completed, yielding positive safety and efficacy results. Areas covered There are two major disorders affecting salivary glands; radiation damage following treatment for head and neck cancers and Sjögren’s syndrome. Salivary gland gene transfer has also been employed in preclinical studies using transgenic secretory proteins for exocrine (upper gastrointestinal tract) and endocrine (systemic) applications. Expert opinion Salivary gland gene transfer is safe and can be beneficial in humans. Applications to treat and prevent radiation damage show considerable promise. A first-in-human clinical trial for the former was recently successfully completed. Studies on Sjögren’s syndrome suffer from an inadequate understanding of its etiology. Proof of concept in animal models has been shown for exocrine and endocrine disorders. Currently, the most promising exocrine application is for the management of obesity. Endocrine applications are limited, as it is currently impossible to predict if systemically required transgenic proteins will be efficiently secreted into the bloodstream. This results from not understanding of how secretory proteins are sorted. Future studies will likely employ ultrasound assisted and pseudotyped adenoassociated viral vector-mediated gene.

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Section: Methodological Advancesmentioning

confidence: 87%

Section: Glandular Disordersmentioning

confidence: 99%

See 1 more Smart Citation

Advances in salivary gland gene therapy – oral and systemic implications

Baum¹,

Alevizos²,

Chiorini³

et al. 2015

Expert Opinion on Biological Therapy

View full text Add to dashboard Cite

show abstract

“…This has been considered as a translational step, as this approach can be used to treat a huge number of patients suffering from dry mouth (xerostomia) due to radiation therapy and autoimmune diseases. A recent clinical trial has advocated the safety of using AQP1 gene therapy for the management of xerostomia patients [72]. Patients undergoing radiotherapy usually develop xerostomia due to irreversible acinar damages.…”

Section: Applications In Dentistrymentioning

confidence: 99%

Gene Therapy: A Paradigm Shift in Dentistry

Siddique

Raza

Ahmed

et al. 2016

Genes

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Gene therapy holds a promising future for bridging the gap between the disciplines of medicine and clinical dentistry. The dynamic treatment approaches of gene therapy have been advancing by leaps and bounds. They are transforming the conventional approaches into more precise and preventive ones that may limit the need of using drugs and surgery. The oral cavity is one of the most accessible areas for the clinical applications of gene therapy for various oral tissues. The idea of genetic engineering has become more exciting due to its advantages over other treatment modalities. For instance, the body is neither subjected to an invasive surgery nor deep wounds, nor is it susceptible to systemic effects of drugs. The aim of this article is to review the gene therapy applications in the field of dentistry. In addition, therapeutic benefits in terms of treatment of diseases, minimal invasion and maximum outcomes have been discussed.

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“…The study procedures were well tolerated over 42 days. Nevertheless, the risks connected to the use of an adenoviral vector impeded an advancement to phase II trials (Wang et al, 2015). The recent successful ultrasound-assisted, nonviral gene transfer of AQP1 to an irradiated swine model may open up new options for human trials.…”

Section: Aqp Gene Replacement Therapymentioning

confidence: 99%