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Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...

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Gene Therapy

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The Progress and Evolving Trends in Nucleic-Acid-Based Therapies.

Yunlong Liu¹, Chunmiao Wang¹, Xiuping Fu¹

¹School of Chemistry and School of Life Sciences, Tiangong University, Tianjin 300387, China.

|March 28, 2025

Summary

Nucleic-acid therapies precisely target genes to suppress protein expression, offering advantages over traditional drugs. Delivery vectors are crucial for enhancing cellular uptake and stability of these promising treatments.

Keywords:

chemical modification clinical use delivery systems nucleic acid drugs’ mechanisms nucleic-acid-based therapeutics trends and challenges

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Area of Science:

Biomedical Science
Molecular Biology
Pharmacology

Background:

Nucleic-acid-based therapies represent a rapidly advancing field in biomedical science.
These therapies utilize DNA or RNA molecules to target specific genes, inhibiting protein expression.
Examples include antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), microRNAs (miRNAs), and messenger RNAs (mRNAs).

Purpose of the Study:

To review various nucleic acid drugs and their mechanisms of action.
To evaluate the current research and clinical applications of nucleic-acid-based therapies.
To discuss future trends and potential challenges in this therapeutic area.

Main Methods:

Literature review of nucleic-acid-based therapies.
Analysis of mechanisms, applications, and delivery systems.
Discussion of pharmacodynamic profiles and clinical research status.

Main Results:

Nucleic-acid therapies offer high specificity and potential for chemical modification, enhancing stability and reducing immunogenicity.
Limited cellular permeability remains a challenge, necessitating effective delivery vectors for intracellular uptake.
Promising pharmacodynamic profiles are driving significant interest in clinical research.

Conclusions:

Nucleic-acid-based therapies demonstrate significant potential for treating a wide range of diseases.
Continued research and development, particularly in delivery systems, are essential for realizing their full therapeutic potential.
These therapies are poised to play a crucial role in future disease management strategies.