Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

Author:

Kim Jinkuk1,Hu Chunguang1,Moufawad El Achkar Christelle1,Black Lauren E.1,Douville Julie1,Larson Austin1,Pendergast Mary K.1,Goldkind Sara F.1,Lee Eunjung A.1,Kuniholm Ashley1,Soucy Aubrie1,Vaze Jai1,Belur Nandkishore R.1,Fredriksen Kristina1,Stojkovska Iva1,Tsytsykova Alla1,Armant Myriam1,DiDonato Renata L.1,Choi Jaejoon1ORCID,Cornelissen Laura1,Pereira Luis M.1,Augustine Erika F.1,Genetti Casie A.1,Dies Kira1,Barton Brenda1,Williams Lucinda1,Goodlett Benjamin D.1,Riley Bobbie L.1,Pasternak Amy1,Berry Emily R.1,Pflock Kelly A.1,Chu Stephen1,Reed Chantal1,Tyndall Kimberly1,Agrawal Pankaj B.1,Beggs Alan H.1,Grant P. Ellen1,Urion David K.1,Snyder Richard O.1,Waisbren Susan E.1,Poduri Annapurna1,Park Peter J.1,Patterson Al1,Biffi Alessandra1,Mazzulli Joseph R.1,Bodamer Olaf1,Berde Charles B.1,Yu Timothy W.1ORCID

Affiliation:

1. From the Divisions of Genetics and Genomics (J.K., C.H., E.A.L., A.S., J.V., R.L.D., J.C., P.B.A., A.H.B., S.E.W., O.B., T.W.Y.), Newborn Medicine (P.B.A., P.E.G.), and Neuroradiology (P.E.G.), the Departments of Neurology (C.M.E.A., D.K.U., A. Poduri), Anesthesiology, Critical Care and Pain Medicine (L.C., C.B.B.), Physical and Occupational Therapy (A. Pasternak, E.R.B., K.A.P.), and Pharmacy (S.C., A. Patterson), the Institutional Centers for Clinical and Translational Research (A.K., B.B., L.W.), and...

Publisher

Massachusetts Medical Society

Subject

General Medicine

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