Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy.

Inherited peripheral neuropathy (IPN) is caused by heterogeneous genetic mutations in more than 100 genes. So far, several treatment options for IPN have been developed and clinically evaluated using small molecules. However, gene therapy-based therapeutic strategies have not been aggressively investigated, likely due to the complexities of inheritance in IPN. Indeed, because the majority of the causative mutations of IPN lead to gainof- function rather than loss-of-function, developing a therapeutic strategy is more difficult, especially considering gene therapy for genetic diseases began with the simple idea of replacing a defective gene with a functional copy. Recent advances in gene manipulation technology have brought novel approaches to gene therapy and its clinical application for IPN treatment. For example, in addition to the classically used gene replacement for mutant genes in recessively inherited IPN, other techniques including gene addition to modify the disease phenotype, modulations of target gene expression, and techniques to edit mutant genes have been developed and evaluated as potent therapeutic strategies for dominantly inherited IPN. In this review, the current status of gene therapy for IPN and future perspectives will be discussed.

Synthesis of Silica-Coated Magnetic Hydroxyapatite Composites for Drug Delivery Applications.

We have prepared a core-shell magnetic silica-coated hydroxyapatite, Fe3O4@SiO2@HAp composite materials for pH-responsive drug delivery applications. Captopril (Cap) and ibuprofen (Ibu) were chosen as model hydrophilic and hydrophobic drugs, respectively. The drugs were encapsulated into the Fe3O4@SiO2@HAp composite via electrostatic interactions with existing amine and carboxylic acid groups during calcium phosphate shell formation. The formation of calcium phosphate shell not only protects the encapsulated drugs from leaching but also controls the release rate of drugs from the composite system depending on various pH conditions. We have tested the release behavior of Cap and Ibu drugs under different pH conditions such as neutral pH (pH 7.4) and acidic pH (pH 5.0), respectively. The study result reveals that the synthesized Fe3O4@SiO2@HAp composite is suitable for release of both water soluble and water insoluble drugs based on a pH-responsive controlled manner.