Wang Hui, Shi Hai-Bo, Yin Shan-Kai
Department of Otolaryngology, Affiliated Sixth People's Hospital of Shanghai Jiao Tong University, Otolaryngology Institute of Shanghai Jiao Tong University, Shanghai 200233, P.R. China.
Exp Ther Med. 2011 Sep;2(5):777-781. doi: 10.3892/etm.2011.296. Epub 2011 Jun 27.
Hair cells in the cochlea can be damaged by various insults, including noise, drugs, infections and presbycusis, which may cause sensorineural hearing loss. Gene therapy is a novel therapeutic technology that, recently, has led to the idea of treating inner ear diseases on a genetic level. Depending on their characteristics, such as a high efficiency in delivery, the capability of specific targeting, multifunctionality, biodegradability, non-toxicity, non-immunogenicity and the capability of limiting DNA degradation, nanovectors, such as polyamidoamine (PAMAM) dendrimers for cellular gene delivery, provide a promising approach to eradicate genetic diseases. They are a new class of highly branched spherical polymers that are highly soluble in aqueous solution. Their unique surface is composed of positively charged primary amine groups which allow them to form stable complexes with plasmid DNA, oligonucleotides, antibodies and drugs. This review provides an overview of the characteristics of PAMAMs which may be used in gene transfer into the cochlea as well as the efforts to improve their transfection efficiency as gene-delivery carriers.
耳蜗中的毛细胞会受到各种损伤，包括噪音、药物、感染和老年性耳聋，这些都可能导致感音神经性听力损失。基因治疗是一种新型治疗技术，最近引发了在基因层面治疗内耳疾病的想法。纳米载体，如用于细胞基因递送的聚酰胺胺（PAMAM）树枝状大分子，因其具有高效递送、特异性靶向能力、多功能性、生物可降解性、无毒性、无免疫原性以及限制DNA降解的能力等特性，为根除遗传性疾病提供了一种有前景的方法。它们是一类新型的高度分支的球形聚合物，在水溶液中高度可溶。其独特的表面由带正电荷的伯胺基团组成，这使得它们能够与质粒DNA、寡核苷酸、抗体和药物形成稳定的复合物。本文综述了可用于将基因导入耳蜗的PAMAM的特性，以及作为基因递送载体提高其转染效率的相关研究。
