Large NIH Grant Supports CRISPR-based Gene Therapy Development for Brain Diseases < Yale School of Medicine

A roughly $40 million National Institutes of Health (NIH) grant awarded to Yale School of Medicine will support the development of a gene-editing platform technology capable of reaching the human brain. The innovative new genome-editing technology, which was developed from the first phase of the NIH Common Fund Somatic Cell Genome Editing (SCGE) program, could potentially lead to treatments or cures for many neurogenetic diseases. If we can prove the concept of this technology in the two diseases we’re studying, we can then apply it to hundreds or thousands of diseases of the brain. Neurogenetic disorders can be devastating, and treatments are scarce. The two-phase grant from the NIH will support research into a novel CRISPR-based gene-editing technology and delivery platform for targeting neurogenetic diseases. The grant will focus on Angelman syndrome and H1-4 syndrome as a proof-of-concept, and could be applicable to many neurogenetic disorders. The new delivery technology, known as

5 Minute Read Share article A roughly $40 million National Institutes of Health (NIH) grant awarded to Yale School of Medicine will support the development of a gene-editing platform technology capable of reaching the human brain. The innovative new genome-editing technology, which was developed from the first phase of the NIH Common Fund Somatic Cell Genome Editing (SCGE) program, could potentially lead to treatments or cures for many neurogenetic diseases. If we can prove the concept of this technology in the two diseases we’re studying, we can then apply it to hundreds or thousands of disea

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