G-quadruplex interacting small molecules and drugs: from bench toward bedside

Area of Science:

• Molecular Biology
• Medicinal Chemistry
• Genetics

Background:

• G-quadruplexes are non-canonical four-stranded DNA structures with emerging roles in cellular processes.
• Evidence suggests G-quadruplexes exist and function within living organisms (in vivo).
• Small molecules have been developed over 20 years to specifically target G-quadruplexes.

Purpose of the Study:

• To demonstrate how small molecule probes have elucidated the biological functions of G-quadruplexes.
• To highlight the potential of G-quadruplexes as therapeutic targets.
• To review the development of G-quadruplex-interacting small molecules as drug candidates.

Main Methods:

• Utilizing specifically designed small molecule probes to investigate G-quadruplex biological roles.
• Analyzing fundamental research linking G-quadruplexes to biological functions and therapeutic potential.
• Discussing the progression of G-quadruplex ligands into potential drug development.

Main Results:

• Small molecule probes have been instrumental in uncovering major biological roles of G-quadruplexes.
• Fundamental research has identified G-quadruplexes as promising therapeutic targets.
• The development of G-quadruplex-interacting small molecules shows potential for anti-carcinogenic activity.

Conclusions:

• Small molecules are key tools for understanding G-quadruplex biology.
• G-quadruplexes represent a significant area for therapeutic intervention, particularly in cancer.
• Targeting G-quadruplexes with small molecules is a viable strategy for drug discovery.