„Glattes Gehirn“ (Lissenzephalie) ist ein Spektrum seltener genetischer Erkrankungen, bei denen das Gehirn seine charakteristischen Falten nicht entwickelt. Derzeit sind keine Behandlungen verfügbar. Eine neue Studie hat jedoch ein Medikament identifiziert, das Lissenzephalie-Fehlbildungen in Gehirnorganoiden verhindert und umkehrt.
https://news.yale.edu/2025/01/01/smooth-brain-disorders-may-share-common-cause-and-potential-treatment
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I’ve linked to the press release in the post above. In this comment, for those interested, here’s the link to the peer reviewed journal article:
https://www.nature.com/articles/s41586-024-08341-9
From the linked article:
‘Smooth brain’ disorders may share a common cause — and potential treatment
Lissencephaly is a spectrum of rare, genetic disorders in which the brain fails to develop its hallmark folds. The disorders are often associated with seizures and intellectual disability and currently there are no available treatments.
A new Yale study, however, has identified a molecular mechanism that underlies some lissencephaly disorders — and a drug that prevents and reverses lissencephaly malformations in organoids (small, three-dimensional replicas of developing brains that allow scientists to study early brain development).
The findings, reported Jan. 1 in Nature, may point to a target for treatment, researchers say.
Amazingly, during the last US election cycle, it was also proven a smooth brained individual could, in fact, be elected to the highest office in the US.
Best of luck for their continued research. I’ve worked in pediatric rehab for decades and one of my very first kids on my caseload had lissencephaly. I haven’t seen another child with that diagnosis since. Being in this field so long one of the most amazing things is seeing prognoses change for the better for so many kids as medical knowledge and technology advances. Hope this happens for lissencephaly.
A smooth brain is more effective because the thoughts are able to glide along the smooth surface easier.
Source: have smooth brain
I want to apologise in advance if I sound insensitive- but I am curious to know if this could be a possible treatment or if this is even possible:
Could a STEM cell treatment possibly ease the symptoms of this genetic disorder?
In the sense that giving the baby a transplant of healthy STEM cells which can go on to create neurons that perhaps could make the intellectual disability not so severe?
I am not sure if we can even lab produce STEM cells, and I also am not certain if this is safe for a baby but I do want to, know if this could be a solution until we find what exactly triggers the gene defect