Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Developing strategies toward safe and effective drug delivery into the central nervous
system (CNS) with improved targeting abilities and reduced off-target effects is crucial. CNS …

Gene vectors are nucleic acids that carry genetic materials or gene editing devices into cells
to exert the sustained production of therapeutic proteins or to correct erroneous genes of the …

Progress in deciphering the genetic architecture of human sensorineural hearing
impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the …

Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

Exosomes are small membrane-bound vesicles of endocytic origin that are actively
secreted. The potential of exosomes as effective communicators of biological signaling in …

Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

Extracellular vesicles (EVs) are cell-derived membrane vesicles virtually secreted by all
cells, including brain cells. EVs are a major term that includes apoptotic bodies …

Historically, small molecules, including steroid hormones and cytokines, have been
attributed a role in paracrine and endocrine signaling, and now include a new player …

For therapeutic materials to be successfully delivered to the heart, several barriers need to
be overcome, including the anatomical challenges of access, the mechanical force of the …