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Desarrollo de Fármacos

Jack Stahl1, Aswathy Joji1, Elianny Perozo Rojas1

  • 1University of Miami Miller School of Medicine, Center for Therapeutic Innovation, Miami, FL, USA.

Alzheimer's & dementia : the journal of the Alzheimer's Association
|December 25, 2025
PubMed
Resumen
Este resumen es generado por máquina.

Los oligonucleótidos modificables en cadena (COSMOs) ofrecen un nuevo enfoque terapéutico para la enfermedad de Alzheimer (EA), reduciendo simultáneamente el péptido amiloide beta (A𝛣) y la proteína tau fosforilada (p-Tau). Esta estrategia de doble acción promete un tratamiento más eficaz para la EA en comparación con las terapias de un solo objetivo.

Palabras clave:
OligonucleótidosTerapia génicaEnfermedad de AlzheimerProteína tauPéptido amiloide betaDesarrollo de fármacos

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Área de la Ciencia:

  • Neuroscience
  • Molecular Biology
  • Pharmacology

Sus antecedentes:

  • Alzheimer's Disease (AD) is characterized by amyloid beta (A𝛣) and phosphorylated tau (p-Tau) accumulation.
  • Current A𝛣-targeting antibodies have limitations, including brain swelling and failure to reduce p-Tau.
  • Oligonucleotide therapeutics show promise, with siRNAs reducing A𝛣 and ASOs reducing p-Tau.

Objetivo del estudio:

  • To develop a novel therapeutic approach for Alzheimer's Disease (AD).
  • To create multi-targeting siRNAs, termed COSMOs, capable of simultaneously reducing both A𝛣 and p-Tau.
  • To evaluate the efficacy and safety of COSMOs in vitro.

Principales métodos:

  • Design, synthesis, and screening of siRNAs targeting amyloid precursor protein (APP) and microtubule-associated protein tau (MAPT).
  • Transfection of human SK-N-AS cells with COSMOs and individual siRNAs.
  • Quantification of mRNA and protein levels of APP, Tau, A𝛣42, and p-Tau181 using qPCR and ELISA.
  • Assessment of cellular viability and COSMO cleavability.

Principales resultados:

  • Top siRNAs and COSMOs demonstrated activity at picomolar concentrations.
  • Co-transfection and COSMO transfection simultaneously reduced A𝛣 and p-Tau levels.
  • APP- and MAPT-targeting COSMOs exhibited lower toxicity than individual siRNAs.
  • COSMOs can be engineered for tunable cleavability.

Conclusiones:

  • No current AD therapeutics effectively reduce both A𝛣 and p-Tau.
  • COSMOs represent a promising new class of therapeutics for AD.
  • COSMOs have the potential to simultaneously target key pathologies in Alzheimer's Disease.