Technologies capable of programmable translation activation offer strategies to develop
therapeutics for diseases caused by insufficient gene expression. Here, we present …

Gene-targeted therapies for genetic neurodevelopmental disorders (NDDs) are becoming a
reality. The Center for Epilepsy and Neurodevelopmental Disorders (ENDD) is currently …

Alternative splicing is a crucial regulator in stem cell biology, intricately influencing the
functions of various biological macromolecules, particularly pre-mRNAs and the resultant …

The patterning of excitatory cortical neurons from human pluripotent stem cells (hPSCs) is a
desired technique for the study of neurodevelopmental disorders, as neurons can be …

Dysfunction of visceral smooth muscle (“visceral myopathy”) impairs bowel, bladder, and
uterine function. Symptoms of this life-threatening condition include massive intestinal …

Visceral myopathies are debilitating conditions characterized by dysfunction of smooth
muscle in visceral organs (bowel, bladder, and uterus). Individuals affected by visceral …

[eng] Cancer treatment has evolved significantly in the last decades and survival ha s
increased steadily. However, the survival rate of aggressive cancers, such as Glioblastoma …