Integrating Machine Learning-Based Approaches into the Design of ASO Therapies

Genes, Год журнала: 2025, Номер 16(2), С. 185 - 185

Опубликована: Фев. 2, 2025

Rare diseases impose a significant burden on affected individuals, caregivers, and healthcare systems worldwide. Developing effective therapeutics for these small patient populations presents substantial challenges. Antisense oligonucleotides (ASOs) have emerged as promising therapeutic approach that targets the underlying genetic cause of disease at RNA level. Several ASOs gained FDA approval treatment conditions, including use in personalized N-of-1 trials. However, despite their potential, often exhibit limited clinical efficacy, optimizing design is complex process influenced by numerous factors. Machine learning-based platforms, eSkip-Finder ASOptimizer, been developed to address challenges predicting optimal ASO sequences chemical modifications enhance efficacy. focuses exon-skipping applications, while ASOptimizer aims optimize degradation. Preliminary vitro results demonstrated predictive power platforms. limitations remain, generalizability alternative gaps consideration all factors influencing efficacy safety. Continued advancements machine learning models, alongside efforts incorporate additional features affecting safety, hold promise field. These platforms potential streamline development, reduce associated costs, improve outcomes, positioning key tool future therapeutics.

Язык: Английский

Preparing for patient-customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases

Опубликована: Май 29, 2024

The process of developing therapies to treat rare diseases is fraught with financial, regulatory, and logistical challenges that have limited our ability build effective treatments. Recently, a novel type therapy called antisense has shown immense potential for the treatment diseases, particularly through single-patient N-of-1 trials. Several been developed recently including landmark study milasen. In response success therapy, Food Drug Administration (FDA) unique guidelines specifically development diseases. This policy change establishes strong foundation future development, addresses some major limitations previously hindered

Язык: Английский

Antisense oligonucleotides in rare neurogenetic disorders

Journal of Genetic Medicine, Год журнала: 2024, Номер 21(2), С. 41 - 50

Опубликована: Дек. 31, 2024

Rapid advancements in genetic testing have significantly improved the diagnosis of rare diseases.However, development targeted therapies has progressed more slowly, leaving most conditions without effective treatment.Because 80% disorders involve nervous system, early intervention is crucial, particularly pediatric patients with progressive conditions.Antisense oligonucleotides (ASOs) emerged as promising therapeutics that offer precise modulation gene expression through RNA targeting, requiring viral delivery systems.These been successful modulating disease trajectories, thereby demonstrating potential precision medicine.Recent innovations ASO chemical modifications and strategies enhanced their safety, stability, tissue specificity, broadening applicability complex neurogenetic disorders.This review explores mechanisms, clinical applications, future ASOs, emphasizes growing role medicine.As diagnostics evolve alongside therapeutics, ASOs are expected to become key pillars for addressing unmet medical needs transforming management previously untreatable disorders.

Язык: Английский