New gene delivery vehicle shows promise for human brain gene therapy

Gene Therapy Berita

New gene delivery vehicle shows promise for human brain gene therapy
Brain TumorBirth DefectsHuman Biology
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In an important step toward more effective gene therapies for brain diseases, researchers have engineered a gene-delivery vehicle that uses a human protein to efficiently cross the blood-brain barrier and deliver a disease-relevant gene to the brain in mice expressing the human protein.

New gene delivery vehicle shows promise for human brain gene therapy ." ScienceDaily. ScienceDaily, 16 May 2024. <www.sciencedaily.comBroad Institute of MIT and Harvard. . New gene delivery vehicle shows promise for human brain gene therapy .

In the first in-human clinical trial, scientists used a novel, skull-implantable ultrasound device to open the blood-brain barrier and repeatedly permeate large, critical regions of the human brain ... The blood-brain barrier is an imposing foe for gene therapy. Formed of cells wedged tightly together, the BBB keeps toxins and pathogens that may be present in the blood from entering brain ...

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