RNA Structure: Past, Future, and Gene Therapy Applications.

Area of Science:

• Molecular Biology
• Genomics
• Biochemistry

Background:

• Ribonucleic acid (RNA) was initially considered a simple intermediary in gene expression.
• RNA is now recognized for its diverse functions, arising from its abundance and complex structures.
• A significant portion of the human genome (70-90%) is transcribed into various functional RNAs.

Purpose of the Study:

• To highlight the critical role of RNA structure in its lifecycle and cellular functions.
• To discuss the challenges and advancements in predicting RNA tertiary and higher-order structures.
• To emphasize the potential of RNA-based applications, particularly in gene therapy.

Main Methods:

• Analysis of RNA structure from primary to quinary levels.
• Comparison with protein structure prediction methods like AlphaFold.
• Integration of machine learning, artificial intelligence, and advanced sequencing techniques.
• Single-cell and intact tissue level structural analyses.

Main Results:

• RNA structure is crucial for its lifecycle, from transcription to decay.
• Predicting complex RNA tertiary and beyond structures remains a significant challenge.
• Emerging technologies offer promising avenues for RNA structure elucidation.

Conclusions:

• RNA's multifaceted roles are intrinsically linked to its intricate structural organization.
• Continued advancements in computational and experimental methods are vital for understanding RNA.
• The progress in RNA structure prediction holds great promise for developing novel gene therapy strategies.