There is growing need for a safe, efficient, specific and non-pathogenic means for delivery of
gene therapy materials. Nanomaterials for nucleic acid delivery offer an unprecedented …

The advent of genome editing has transformed the therapeutic landscape for several
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …

Genome editing offers promising solutions to genetic disorders by editing DNA sequences
or modulating gene expression. The clustered regularly interspaced short palindromic …

Noninvasive aerosol inhalation is an established method of drug delivery to the lung, and
remains a desirable route for nucleic‐acid‐based therapeutics. In vitro transcribed (IVT) …

Gene-based therapy is the intentional modulation of gene expression in specific cells to treat
pathological conditions. This modulation is accomplished by introducing exogenous nucleic …

Gene therapy is a promising strategy for the treatment of monogenic disorders. Non-viral
gene delivery systems including lipid-based DNA therapeutics offer the opportunity to …

Since the elucidation of the molecular structure of nucleic acids and understanding of their
role in encoding and translating genetic information into biological function, 1 the knowledge …

Nucleic acid therapy can achieve lasting and even curative effects through gene
augmentation, gene suppression, and genome editing. However, it is difficult for naked …

Gene therapy using nucleic acids has many clinical applications for the treatment of
diseases with a genetic origin as well as for the development of innovative vaccine …

Gene therapy is the process of introducing foreign genomic materials into host cells to elicit
a therapeutic benefit. Although initially the main focus of gene therapy was on special …