Using a highly versatile form of CRISPR gene editing, researchers successfully restored vision in mice with retinitis pigmentosa.
CRISPR gene editing created the G795A amino acid which was introduced to microglia derived from human stem cells. Researchers were able to transplant the donor microglia immune cells into humanized rodent models while administering an FDA-approved cancer drug called pexidartinib. The inclusion of the amino acid cause the donated microglia to thrive and resist the drug, while the host microglia died. The findings open the door for new methods of using microglia to treat a range of neurodegenerative disorders.
Researchers have developed a new family of nano-scale capsules capable of carrying CRISPR gene editing tools to different organs of the body before harmlessly dissolving. The capsules were able to enter the brains of mice and successfully edit a gene associated with Alzheimer's disease.
A new stem cell therapy approach eliminates established brain tumors and provides long-term immunity, training the immune system to prevent cancer from returning.
Using CRISPR gene editing to directly target RNA, researchers eliminated toxic protein buildup associated with Huntington's disease while not disrupting other human genes.
New research in cloned pigs with a mutation of the SORL1 sheds light on Alzheimer's development. The findings could pave the way for new treatments for the neurodegenerative disorder.
Using CRISPR gene editing, researchers were able to control microglia and reverse their toxic state associated with Alzheimer's disease, and put them back on track.
Gene editing could be a potential new treatment for anxiety and alcohol use disorder in adults who indulged in binge drinking as adolescence.
Using gene editing to disrupt a gene in the thalamus that codes for a protein that binds to GABA boosted the activity of delta waves and promoted deep sleep in mouse models.
Researchers successfully transplanted genetically modified, clinical-grade pig kidneys into a brain-dead human, replacing their native kidneys. The results demonstrate how xenotransplantation could help address organ shortages.
Gene editing may provide hope for the treatment of Fragile X, the leading genetic cause of autism.
The dSlo2 channel appears to play a role in suppressing hyperactivity in the brain that causes epileptic seizures.
