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Preclinical development consists of a series of tests that ensure the safety and efficacy of a new therapeutic compound before it is tested in humans. There are four main phases to this process. First, safety pharmacology tests are conducted to ensure the drug does not produce any acutely harmful effects. These tests examine parameters such as bronchoconstriction, cardiac dysrhythmias, blood pressure changes, and ataxia. Next, preliminary toxicological testing is performed to determine the...

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Drug Repurposing Hypothesis Generation Using the "RE:fine Drugs" System

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Desarrollo de fármacos

Malik White^1,2, Rikke Han Kofoed^3,4,5, Chinaza Lilian Dibia^1,2

¹University of Toronto, Toronto, ON, Canada.

Alzheimer's & dementia : the journal of the Alzheimer's Association

|December 26, 2025

Resumen

La combinación de vectores de virus adenoasociados (VAA) intravenosos con ultrasonido enfocado (FUS) mejora la entrega de genes a través de la barrera hematoencefálica. Este método no invasivo permite la terapia génica precisa o generalizada para trastornos cerebrales.

Palabras clave:

terapia génica ultrasonido enfocado entrega de genes barrera hematoencefálica enfermedades neurodegenerativas VAA Alzheimer Parkinson

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Área de la Ciencia:

Neurociencia; Terapia Génica; Imagenología Médica

Sus antecedentes:

Los virus adenoasociados recombinantes (VAA) son prometedores para los trastornos cerebrales, pero enfrentan desafíos con la entrega invasiva y la penetración de la barrera hematoencefálica (BHE).; La entrega intravenosa (i.v.) de VAA es ineficiente debido a la BHE, lo que limita el alcance terapéutico.; Las nuevas variantes de VAA y el ultrasonido enfocado guiado por MRI (FUS) ofrecen soluciones potenciales para mejorar la transfección cerebral.

Objetivo del estudio:

Investigar la eficacia combinada de los VAA intravenosos que penetran la BHE y el FUS para optimizar la entrega de genes en el cerebro.; Lograr la transfección génica generalizada y específica de la región para el tratamiento de enfermedades neurodegenerativas.

Principales métodos:

Se inyectaron en ratones C57BL/6 VAA-PHP.V1.CAG.TdTomato (V1) y AAV9.CAG.EYFP por vía intravenosa.; Se aplicó ultrasonido enfocado (FUS) para dirigirse a varias regiones del cerebro después de la inyección.; Se utilizó inmunohistoquímica y microscopía confocal para el análisis 3 semanas después de la administración.

Principales resultados:

El FUS mejoró significativamente la entrega génica dirigida tanto para los vectores AAV9 como V1.; Los vectores V1 demostraron una expresión global mantenida en el cerebro junto con un enriquecimiento local mejorado por FUS.; El enfoque combinado ofrece una estrategia no invasiva para la terapia génica precisa (AAV9) o generalizada (V1).

Conclusiones:

Esta estrategia combinada de VAA y FUS mejora la eficiencia de la entrega de genes a través de la BHE.; El enfoque tiene potencial para mejorar los tratamientos de trastornos neurodegenerativos como la enfermedad de Alzheimer y Parkinson.