A G-quadruplex (G4)
is a four-stranded nucleic acid secondary structure
maintained by Hoogsteen hydrogen bonds established between four guanines.
Experimental studies and bioinformatics predictions support the hypothesis
that these structures are involved in different cellular functions
associated with both DNA and RNA processes. An increasing number of
diseases have been shown to be associated with abnormal G4 regulation.
Here, we describe the existence of G4 and then discuss G4-related
pathogenic mechanisms in neurodegenerative diseases and the viral
life cycle. Furthermore, we focus on the role of G4s in the design
of antiviral therapy and neuropharmacology, including G4 ligands,
G4-based aptamers, G4-related proteins, and CRISPR-based sequence
editing, along with a discussion of limitations and insights into
the prospects of this unusual nucleic acid secondary structure in
therapeutics. Finally, we highlight progress and challenges in this
field and the potential G4-related research fields.