Journal of Virology & Antiviral ResearchISSN: 2324-8955

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Commentary, J Virol Antivir Vol: 12 Issue: 2

RNA Interference (RNAi) Therapies for Rare Genetic Disorders: A Promising Frontier in Precision Medicine

Lanier Corbin*

1Department of Pathology, Oklahoma University Health Sciences Center, Oklahoma City, USA

*Corresponding Author: Lanier Corbin,
Department of Pathology, Oklahoma University Health Sciences Center, Oklahoma City, USA

Received date: 29 May, 2023, Manuscript No. JVA-23-107857;

Editor assigned date: 31 May, 2023, PreQC No. JVA-23-107857 (PQ);

Reviewed date: 15 June, 2023, QC No. JVA-23-107857;

Revised date: 22 June, 2023, Manuscript No. JVA-23-107857 (R);

Published date: 29 June, 2023, DOI: 10.4172/2324-8955.1000679

Citation: Corbin L (2023) RNA Interference (RNAi) Therapies for Rare Genetic Disorders. J Virol Antivir Res 12:2.




Rare genetic disorders pose significant challenges to patients and their families due to their often severe and life-threatening nature. Traditional treatment approaches have been limited to managing symptoms, leaving a significant unmet medical need for targeted and curative therapies. The advent of RNA interference (RNAi) technology offers new hope in addressing the root cause of these genetic disorders. RNAi therapies can specifically silence disease-causing genes, potentially halting disease progression and improving patients' quality of life. In this commentary article, we highlight the recent advances and potential applications of RNAi-based treatments for rare genetic disorders, underscoring the transformative potential of this precision medicine approach.

RNAi mechanism and therapeutic potential

RNAi is a natural cellular process that regulates gene expression through the degradation or translational inhibition of specific messenger RNA (mRNA) molecules. This process is initiated by small RNA molecules, including Small Interfering RNAs (siRNAs) and microRNAs (miRNAs), which guide the RNA-Induced Silencing Complex (RISC) to target mRNAs. Upon binding, RISC cleaves or suppresses the target mRNA, preventing the synthesis of the corresponding protein. The specificity and versatility of RNAi make it an attractive therapeutic approach for genetic disorders caused by single gene mutations. By delivering synthetic siRNAs or miRNAs that target disease-causing genes, RNAi therapies can effectively silence the expression of faulty proteins, correcting the underlying molecular defect.

Recent advances in RNAi therapies

Over the past decade, RNAi technology has witnessed remarkable progress, leading to the development of several RNAi-based therapies approved by regulatory agencies. Notably, patisiran and givosiran have received approval for the treatment of rare genetic disorders, such as hereditary Transthyretin-Mediated Amyloidosis (hATTR) and Acute Hepatic Porphyria (AHP), respectively. Patisiran is an RNAi therapeutic that targets and silences the Transthyretin (TTR) gene in hATTR patients, preventing the accumulation of amyloid deposits in tissues. Givosiran, on the other hand, targets and silences Amino Levulinic Acid Synthase 1 (ALAS1) mRNA in AHP patients, reducing the production of toxic heme intermediates. These approved RNAi therapies have demonstrated not only impressive efficacy but also a favorable safety profile, validating the potential of RNAi technology as a viable treatment option for rare genetic disorders.

Expanding Applications in Precision Medicine

Conditions such as Duchenne Muscular Dystrophy (DMD), Huntington's disease, Spinal Muscular Atrophy (SMA), and familial hypercholesterolemia are among the many disorders that stand to benefit from RNAi-based treatments. In DMD, for instance, RNAi therapies targeting the dystrophin gene could hold the key to restoring functional dystrophin expression in muscle cells. By addressing the primary genetic defect responsible for muscle degeneration, RNAibased treatments have the potential to significantly improve the prognosis and functional outcomes for DMD patients. In the case of SMA, the FDA-approved RNAi therapeutic nusinersen has already demonstrated significant clinical benefits in increasing motor function and prolonging survival in infants and children with SMA. Ongoing research is focused on optimizing the delivery and efficacy of RNAi therapies to further enhance outcomes for SMA patients of all ages.

Challenges and Future Perspectives

Despite the considerable progress made in RNAi therapies, challenges remain in their widespread implementation for rare genetic disorders. Delivery of RNAi molecules to target tissues and cells remains a major obstacle, particularly for disorders affecting organs with complex barriers, such as the central nervous system. Additionally, the long-term safety and durability of RNAi therapies need to be carefully evaluated to ensure sustained therapeutic effects and minimize potential off-target effects. In the future, advancements in nanotechnology and gene editing technologies, along with improved understanding of disease mechanisms, will likely address these challenges. Targeted delivery systems, such as lipid nanoparticles or viral vectors, could enhance the tissue specificity and efficiency of RNAi-based treatments.


RNAi technology has emerged as a transformative therapeutic approach for rare genetic disorders, offering hope for targeted and curative treatments. With the successful approval of RNAi therapies for hATTR and AHP, the field is poised to expand rapidly to encompass a broader range of genetic diseases. As research continues to progress, RNAi therapies are expected to usher in a new era of precision medicine, transforming the lives of patients and their families affected by rare genetic disorders. Collaborative efforts among researchers, clinicians, and industry stakeholders are critical to overcoming existing challenges and unlocking the full potential of RNAi-based treatments in precision medicine.

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