Motoneuron diseases (MNDs) are neurodegenerative conditions associated with death of
upper and/or lower motoneurons (MNs). Proteostasis alteration is a pathogenic mechanism …

Motor neuron diseases (MNDs) include a broad group of diseases in which
neurodegeneration mainly affects upper and/or lower motor neurons (MNs). Although the …

RNA splicing regulation has revolutionized the treatment of challenging diseases.
Neuroendocrine cancers, including small cell lung cancer (SCLC) and neuroendocrine …

Recombinant adeno-associated virus (rAAV) has been developed as a safe and effective
gene delivery vehicle to treat rare genetic diseases. This study aimed to establish a novel …

Abstract Glioblastomas (GBM), constituting around 50% of gliomas, represent the most
aggressive and malignant form of brain tumors, characterized by significant heterogeneity …

Fetal gene therapy was first proposed toward the end of the 1990s when the field of gene
therapy was, to quote the Gartner hype cycle, at its “peak of inflated expectations.” Gene …

Antecedentes. La atrofia muscular espinal es una patología neurológica degenerativa
hereditaria. Causa una degeneración paulatina de las motoneuronas de la asta anterior …