Caspase 3 activation is a hallmark of cell death and there is a strong correlation between elevated protease activity and evolving pathology in neurodegenerative disease, such as amyotrophic lateral sclerosis (ALS). At the cellular level, ALS is characterized by protein aggregates and inclusions, comprising the RNA binding protein TDP-43, which are hypothesized to trigger pathogenic activation of caspase 3. However, a growing body of evidence indicates this protease is essential for ensuring cell viability during growth, differentiation and adaptation to stress.
View Article and Find Full Text PDFObjectives: Evaluate the experiences and perceptions of patients participating in a simulated clinical trial and identify ways to enhance future patient-centric trial designs.
Design: International, multicentre, non-interventional, virtual clinical trial visits with patient debriefs and advisory boards.
Setting: Virtual clinic visits and accompanying advisory boards.
The induction of lineage-specific gene programs are strongly influenced by alterations in local chromatin architecture. However, key players that impact this genome reorganization remain largely unknown. Here, we report that the removal of the special AT-rich binding protein 2 (SATB2), a nuclear protein known to bind matrix attachment regions, is a key event in initiating myogenic differentiation.
View Article and Find Full Text PDFBackground: Generalized pustular psoriasis (GPP) is a rare disease characterized by episodic worsening (flares). Knowledge of the burden of GPP and the experience of affected individuals is limited.
Aims: To conduct a survey of people living with GPP to understand how they experience GPP flares, which therapies they have received and are receiving, and how GPP impacts their activities of daily living.