Non-viral vehicles hold therapeutic promise in advancing the delivery of a variety of cargos
in vitro and in vivo, including small molecule drugs, biologics, and especially nucleic acids …

Highlights•Luxturna, a viral gene therapy product, is approved for rare Leber's congenital
amaurosis.•Ocular gene therapies are now being developed for non-inherited retinal …

The broad clinical application of mRNA therapeutics has been hampered by a lack of
delivery vehicles that induce protein expression in extrahepatic organs and tissues …

Targeted protein degradation (TPD) has emerged as a promising therapeutic strategy. Most
TPD technologies use the ubiquitin–proteasome system, and are therefore limited to …

Since first reported, RNA interference (RNAi) has become a widely used tool for cellular
genetic knockdown. However, RNA instability and susceptibility to enzymatic degradation …

Implementation of gene editing technologies such as CRISPR/Cas9 in the manufacture of
novel cell-based therapeutics has the potential to enable highly-targeted, stable, and …

CRISPR/Cas technologies have advanced dramatically in recent years. Many different
systems with new properties have been characterized and a plethora of hybrid CRISPR/Cas …

Targeted protein degradation (TPD) has emerged as an effective therapeutic strategy for a
wide range of diseases; however, the blood-brain barrier (BBB) limits access of degraders …

Weak polyelectrolytes are relevant for a wide range of fields; in particular, they have been
investigated as “smart” materials for chemical separations and drug delivery. The charges …

Mammalian cells express a variety of nucleic acid sensors as one of the first lines of defense
against infection. Despite extensive progress in the study of sensor signaling pathways …