Summary: Researchers have developed a breakthrough system to deliver large therapeutic molecules into the brain, overcoming the challenges of the blood-brain barrier. The innovative blood-brain barrier-crossing conjugate (BCC) platform utilizes a biological process called γ-secretase-mediated transcytosis to safely transport drugs like oligonucleotides and proteins into the central nervous system via intravenous injection.
In mouse models and human brain tissue, the system effectively silenced harmful genes linked to diseases such as ALS and Alzheimer’s without causing significant side effects. This advancement could revolutionize treatments for neurological and psychiatric disorders, solving a critical challenge in brain research.
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