Research News BDSRA Australia, together with BDSRA Foundation, is proud to have been involved in this groundbreaking new research!
We know that individuals with Batten disease often suffer from severe gastrointestinal issues. This new study may have uncovered why: the neurons controlling the gut (the enteric nervous system) degenerate over time.
In a study using mouse models of CLN1 and CLN2 disease, the team found that neurons in the gut progressively die, leading to slower digestion and worsening symptoms with age. Similar pathology was evident in tissue from a child with CLN1 disease. Remarkably, they discovered that gene therapy given early in life could protect these gut neurons in mice with CLN1 and CLN2 disease, improving digestion and even extending lifespan.
Notably, this exciting study - and the new therapeutic possibilities it unveils - was directly inspired by Batten families, who highlighted the significant impact of gastrointestinal issues on quality of life and the need for more research in this area.
Congratulations to Prof. Jonathan Cooper, @batten_psdl, Dr Ewa Ziolkowska, our own Dr Ineka Whiteman, and the entire team.
To access the article head to: https://www.science.org/doi/10.1126/scitranslmed.adj1445
Read more here about the research journey here: https://medicine.washu.edu/.../fatal.../...