Ribosome Biogenesis, Function, and Therapeutic Targeting: From Cancer to Genetic Disease

Session

Pharmaceutical and Natural Sciences

Description

Ribosome biogenesis and function are vital for cellular protein synthesis and are profoundly altered in cancer and various genetic diseases. Enhanced ribosome production and mutations in ribosomal proteins contribute to malignancy, positioning the ribosome as a promising therapeutic target. Advances in understanding ribosomal regulation, including the pivotal tumor suppressor p53, nonsense mutation readthrough therapies, and the impact of alternative splicing, have revealed new strategies for intervention in both cancer and rare ribosomopathies. Pharmacological inhibition of ribosome biogenesis, selective ribosome editing, and modulation of post-translational modifications offer potential to disrupt tumor proliferation or restore functional proteins in genetic disorders. Technologies like RiboScreen™ and targeted ligands exemplify innovative approaches to augment protein production or correct genetic defects, while studies also highlight the influence of oxidative stress and reactive oxygen species on ribosomal integrity and disease progression. The integration of ribosometargeted therapies with insights into metabolic rewiring, stress responses, and epigenetic regulation underscores the need for precise, context-aware interventions. Ongoing research into ribosomal protein roles and modifications promises to advance both diagnostic practices and therapeutic development for cancer, inherited disorders, and metabolic disease.

Keywords:

Ribosome, RiboScreen™, p53, NMD therapies

Proceedings Editor

Edmond Hajrizi

ISBN

978-9951-982-41-2

Location

UBT Lipjan, Kosovo

Start Date

25-10-2025 9:00 AM

End Date

26-10-2025 6:00 PM

DOI

10.33107/ubt-ic.2025.344

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Oct 25th, 9:00 AM Oct 26th, 6:00 PM

Ribosome Biogenesis, Function, and Therapeutic Targeting: From Cancer to Genetic Disease

UBT Lipjan, Kosovo

Ribosome biogenesis and function are vital for cellular protein synthesis and are profoundly altered in cancer and various genetic diseases. Enhanced ribosome production and mutations in ribosomal proteins contribute to malignancy, positioning the ribosome as a promising therapeutic target. Advances in understanding ribosomal regulation, including the pivotal tumor suppressor p53, nonsense mutation readthrough therapies, and the impact of alternative splicing, have revealed new strategies for intervention in both cancer and rare ribosomopathies. Pharmacological inhibition of ribosome biogenesis, selective ribosome editing, and modulation of post-translational modifications offer potential to disrupt tumor proliferation or restore functional proteins in genetic disorders. Technologies like RiboScreen™ and targeted ligands exemplify innovative approaches to augment protein production or correct genetic defects, while studies also highlight the influence of oxidative stress and reactive oxygen species on ribosomal integrity and disease progression. The integration of ribosometargeted therapies with insights into metabolic rewiring, stress responses, and epigenetic regulation underscores the need for precise, context-aware interventions. Ongoing research into ribosomal protein roles and modifications promises to advance both diagnostic practices and therapeutic development for cancer, inherited disorders, and metabolic disease.