The Bespoke Gene Therapy Consortium: facilitating development of AAV gene therapies for rare diseases

P. J. Brooks,Timothy M. Miller, Frédéric Revah,Junghae Suh, Bradley R. Garrison, Lawrence C. Starke, Timothy K. MacLachlan, Edward G. Neilan, Gopa Raychaudhuri, Sadik H. Kassim, Jean Dehdashti, Joni L. Rutter

Nature Reviews Drug Discovery（2024）

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摘要

public–private partnership managed by the FNIH aims to address biological, manufacturing and regulatory challenges to the development of gene therapies for rare diseases.

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