Viral vectors represent a bottleneck in the manufacturing of cellular therapies.
Electroporation has emerged as an approach for non-viral transfection of primary cells, but …

Inner ear gene therapy has recently effectively restored hearing in neonatal mice, but it is
complicated in adulthood by the structural inaccessibility of the cochlea, which is embedded …

Background aims Next-generation immune cell therapy products will require complex
modifications using engineering technologies that can maintain high levels of cell …

Nanoparticle-based CRISPR/Cas9 delivery systems hold great promise for specific and
precise treatment of genetic disorder diseases. Herein, we developed a DNA nanoflower …

Noise‐induced hearing loss (NIHL) poses a significant burden on not only the economics of
health care but also the quality of life of an individual, as we approach an unprecedented …

The-state-of-art CRISPR/Cas9 is one of the most powerful among the approaches being
developed to rescue fundamental causes of gene-based inheritable diseases. Several …

Abstract Adeno-associated virus (AAV)-mediated gene delivery systems have been shown
to be effective tools for gene manipulation in the inner ear. For example, hair cells (HCs) and …

Clustered regularly interspaced palindromic repeats (CRISPR) is a gene editing tool with
tremendous therapeutic potential. Recently, ribonucleoprotein (RNP) complex‐based …

Mutations in microRNA-96 (MIR96) cause dominant delayed onset hearing loss DFNA50
without treatment. Genome editing has shown efficacy in hearing recovery by intervention in …

Safely and efficiently controlling gene expression is a long-standing goal of biomedical
research, and the recently discovered bacterial CRISPR/Cas system can be harnessed to …