Study unveils new way to unlock blood-brain barrier - Video
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Overview
Researchers at the Icahn School of Medicine at Mount Sinai have developed an innovative approach-demonstrated in mouse models and isolated human brain tissue—to safely and effectively deliver therapeutics into the brain, providing new possibilities for treating a wide range of neurological and psychiatric diseases.
Published in the online issue of Nature Biotechnology, the study introduces a blood-brain barrier-crossing conjugate (BCC) system, designed to overcome the protective barrier that typically blocks large biomolecules from reaching the central nervous system (CNS).
The blood-brain barrier-crossing conjugate (BCC) system, designed to overcome the protective barrier that typically blocks large biomolecules platform takes advantage of a specialized biological process called γ-secretase-mediated transcytosis to deliver large therapeutic molecules, like oligonucleotides and proteins, directly into the brain through a simple intravenous injection.
The study showed that when the researchers injected a compound called BCC10 linked to specialized genetic tools known as antisense oligonucleotides into mice, it successfully reduced the activity of harmful genes in the brain. In a transgenic mouse model of ALS, the treatment significantly lowered levels of the disease-causing gene called Sod1 and its associated protein.
BCC10 proved to be highly effective at delivering these genetic tools to the brain, improving their ability to silence harmful genes in different models and even in samples of excised human brain tissue studied in the laboratory. Importantly, the treatment was well tolerated in mice, causing little or no damage to major organs at the tested doses, say the investigators.
Reference: Wang, C., Wang, S., Xue, Y. et al. Intravenous administration of blood–brain barrier-crossing conjugates facilitate biomacromolecule transport into central nervous system. Nat Biotechnol (2024). https://doi.org/10.1038/s41587-024-02487-7
Speakers
Dr. Bhumika Maikhuri
BDS, MDS
Dr Bhumika Maikhuri is a Consultant Orthodontist at Sanjeevan Hospital, Delhi. She is also working as a Correspondent and a Medical Writer at Medical Dialogues. She completed her BDS from Dr D Y patil dental college and MDS from Kalinga institute of dental sciences. Apart from dentistry, she has a strong research and scientific writing acumen. At Medical Dialogues, She focusses on medical news, dental news, dental FAQ and medical writing etc.
