Experimental approaches to address the mechanisms of MND

20 Outubro 2021, 11:30 • Margarida Henriques da Gama Carvalho

Research seminar

Spinal Muscular Atrophy

20 Outubro 2021, 09:30 • Margarida Henriques da Gama Carvalho

Overview of Spinal Muscular Atrophy (SMA) – from disease genetics and mechanisms to therapeutic developments

SMA as an unusual perspective on Neurodegeneration – from loss-of-function to absence of aggregation phenotypes

Costs and values of gene therapy approaches for rare disorders 

Making sense of multiple disease genes – common phenotypes – diseases as molecular network disfunctions

Experimental approaches to address the role of RNA metabolism in ALS

13 Outubro 2021, 11:30 • Margarida Henriques da Gama Carvalho

Research seminar by Prof. Dora Brites, FFUL

"Neuroinflammation and miRNA-targeted therapeutics in Amyotrophic Lateral Sclerosis"

RNA metabolism in ND – FTD and ALS

13 Outubro 2021, 09:30 • Margarida Henriques da Gama Carvalho

RNA metabolism and neurodegeneration – connections and functional significance

Amyotrophic Lateral Sclerosis (ALS):  symptoms, presentation, genetics, causal genes and relation to RNA metabolism

Neuropathology of ALS – a cell autonomous, protein aggregation disease?

The prion-like properties of RNA binding proteins and the potential role of RNA in liquid-phase separation

Genetics in Neurodegeneration

6 Outubro 2021, 11:30 • Margarida Henriques da Gama Carvalho

Critical reading and group activity