A recent study published in Cell Stem Cell sheds new light on how transplanted human neurons can repair damaged brain circuits after injury. In this research, scientists transplanted human embryonic stem cell (ESC)-derived cortical progenitors into the motor cortex of mice with ischemic brain injury. Remarkably, these cells not only survived and matured, but also reconstituted key neural circuits in the adult brain.
image: freepik
This advance could change how we approach genetic diseases like cystic fibrosis and haemophilia, where thousands of different mutations can cause the same condition. Instead of creating separate treatments for each mutation variant, evoCAST might allow doctors to use a single therapy that inserts a healthy gene, regardless of which specific mutation a patient has.
The system grew from studying bacterial “jumping genes” that researchers adapted for human cells through a process mimicking evolution. Using the PACE technique to speed up this evolution through hundreds of generations, the team boosted editing efficiency from minimal levels to 30-40% of cells, good enough for many real-world applications.
Beyond treating inherited diseases, evoCAST could improve CAR T-cell therapies for cancer and help researchers who need precise genetic modifications. While getting these tools into the right cells remains challenging, this technology brings us closer to gene therapies that could help patients with previously difficult-to-treat genetic conditions.
Research article: Transcriptional code for circuit integration in the injured brain by transplanted human neurons
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