Are we prepared to deliver gene‐targeted therapies for rare diseases?

Yu TW, Kingsmore SF, Green RC, MacKenzie T, Wasserstein M, Caggana M, Gold NB, Kennedy A, Kishnani PS, Might M, Brooks PJ, Morris JA, Parisi MA, Urv TK
American Journal of Medical Genetics Part C: Seminars in Medical Genetics January 2023  

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