David R. Liu: A Medical Milestone in Patient-Specific Gene Editing – From design to dosing in 7 months
David R. Liu, Core Institute Member and Vice-Chair of the Faculty at Broad Institute of MIT and Harvard, shared a post on X:
“In a medical milestone, a customized base editor was developed, characterized in human and mouse cells, tested in mice, studied for safety in non-human primates, cleared by US FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe, rapidly progressing genetic disease… all in an astounding 7 months.
Best of all, the infant patient shows apparent benefit. Congratulations to Kiran Musunuru, Rebecca Ahrens-Nicklas, and other team members for this heroic and inspiring effort, which has implications for the hundreds of millions of patients that suffer from thousands of genetic diseases.”
Title: Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease
Journal: The NEJM
Authors: Kiran Musunuru, S.A. Grandinette, X. Wang, T.R. Hudson, K. Briseno, A.M. Berry, J.L. Hacker, A. Hsu, R.A. Silverstein, L.T. Hille, A.N. Ogul, N.A. Robinson-Garvin, J.C. Small, S. McCague, S.M. Burke, C.M. Wright, S. Bick, V. Indurthi, S. Sharma, M. Jepperson, C.A. Vakulskas, M. Collingwood, K. Keogh, A. Jacobi, M. Sturgeon, C. Brommel, E. Schmaljohn, G. Kurgan, T. Osborne, H. Zhang, K. Kinney, G. Rettig, C.J. Barbosa, S.C. Semple, Y.K. Tam, C. Lutz, L.A. George, B.P. Kleinstiver, D.R. Liu, K. Ng, S.H. Kassim, P. Giannikopoulos, M.-G. Alameh, Fyodor D. Urnov, R.C. Ahrens-Nicklas
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