A recent publication in Advanced Experimental Medicine and Biology has pointed to the ways that stem cell technology is not only helping us to develop promising therapeutics for a number of diseases, but it is also helping us advance our understanding of a number of brain disorders.
The authors point to well-known diseases like Parkinson’s Disease and Alzheimer’s Disease – that, despite much attention, funding, and research, are still not preventable or fully curable diseases. They also point to less well-known diseases like fragile X syndrome, Angelman syndrome, Prader-Willi syndrome, and Rett syndrome.
Fragile X syndrome affects more males than females and delayed speech and language development. It is characterized by learning disabilities and deficits in cognition.
Similarly, Angelman syndrome, which is rare, is associated with developmental delays. However, those affected by this syndrome also suffer from balance disturbances, seizures, and difficulties with walking.
Prader-Willi syndrome leads to weak muscle tone and affects many parts of the body. Physical development is therefore what tends to be delayed in this syndrome. Type 2 Diabetes and obesity are common complications.
Patients with Rett syndrome are almost always female and display repetitive hand motions constantly while they are awake. These patients experience difficulties across a spectrum of functions, including speaking, eating, walking, and breathing.
All these diseases that authors of this publication discuss have genetic underpinnings, and, according to the authors, can be investigated in new ways thanks to stem cell technologies. What stem cell technology affords is an enormous source of brain cells and their precursors that can be used to help model these diseases and to observe how different factors affect aspects of the disease.
With better models, tests of therapeutics become more robust as well. The hope is that eventually, stem cells will enable us to understand (epi)genetic brain disorders and also develop ways to effectively prevent or treat them.