Advances in salivary gland gene therapy - oral and systemic implications

被引:24
作者
Baum, Bruce J. [1 ]
Alevizos, Ilias [1 ]
Chiorini, John A. [1 ]
Cotrim, Ana P. [1 ]
Zheng, Changyu [1 ]
机构
[1] Natl Inst Dent & Craniofacial Res, NIH, Mol Physiol & Therapeut Branch, Bethesda, MD 20892 USA
关键词
gene therapy; non-viral gene transfer; radiation damage; salivary glands; secretory proteins; Sjogren's syndrome; viral vectors; ADENOVIRAL-MEDIATED TRANSFER; PIG PAROTID-GLANDS; TRANSGENIC SECRETORY PROTEINS; INCREASED FLUID SECRETION; RAT SUBMANDIBULAR-GLANDS; CELLS IN-VITRO; SJOGRENS-SYNDROME; GROWTH-HORMONE; EPITHELIAL-CELLS; ENDOCRINE SECRETION;
D O I
10.1517/14712598.2015.1064894
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
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 Sjogren's syndrome (SS). 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 SS 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 how secretory proteins are sorted. Future studies will likely employ ultrasound-assisted and pseudotyped adeno-associated viral vector-mediated gene transfer.
引用
收藏
页码:1443 / 1454
页数:12
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