Polyamidoamine dendrimers as gene delivery carriers in the inner ear: How to improve transfection efficiency.

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.