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

Affiliations

• ¹ Division of Genetics and Genomics, Harvard Medical School, Boston, Massachusetts, USA.
• ² Rady Children's Institute for Genomic Medicine, San Diego, California, USA.
• ³ Department of Genetics-Medicine, Brigham and Women's Hospital, Boston, Massachusetts, USA.
• ⁴ Department of Surgery and the Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, San Francisco, California, USA.
• ⁵ Division of Pediatric Genetic Medicine, Children's Hospital at Montefiore, New York, New York, USA.
• ⁶ Division of Genetics, New York State Department of Health, Albany, New York, USA.
• ⁷ Massachusetts General Hospital Department of Pediatrics, Boston, Massachusetts, USA.
• ⁸ EveryLife Foundation for Rare Diseases, Washington, District of Columbia, USA.
• ⁹ Department of Pediatrics, Duke University, Durham, North Carolina, USA.
• ¹⁰ Hugh Kaul Precision Medicine Institute, University of Alabama at Birmingham, Birmingham, Alabama, USA.
• ¹¹ Office of Rare Disease Research, National Center for Advancing Translational Science, National Institutes of Health, Bethesda, Maryland, USA.
• ¹² Division of Neuroscience, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland, USA.
• ¹³ Intellectual and Developmental Disabilities Branch, Eunice Kennedy Shriver, National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.