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

Yu, Timothy W; Kingsmore, Stephen F; Green, Robert C; MacKenzie, Tippi; Wasserstein, Melissa; Caggana, Michele; Gold, Nina B; Kennedy, Annie; Kishnani, Priya S; Might, Matthew; Brooks, Phillip J; Morris, Jill A; Parisi, Melissa A; Urv, Tiina K

Yu, Timothy W; Kingsmore, Stephen F; Green, Robert C; MacKenzie, Tippi; Wasserstein, Melissa; Caggana, Michele; Gold, Nina B; Kennedy, Annie; Kishnani, Priya S; Might, Matthew; Brooks, Phillip J; Morris, Jill A; Parisi, Melissa A; Urv, Tiina K.

Afiliação

Yu TW; Division of Genetics and Genomics, Harvard Medical School, Boston, Massachusetts, USA.
Kingsmore SF; Rady Children's Institute for Genomic Medicine, San Diego, California, USA.
Green RC; Department of Genetics-Medicine, Brigham and Women's Hospital, Boston, Massachusetts, USA.
MacKenzie T; Department of Surgery and the Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, San Francisco, California, USA.
Wasserstein M; Division of Pediatric Genetic Medicine, Children's Hospital at Montefiore, New York, New York, USA.
Caggana M; Division of Genetics, New York State Department of Health, Albany, New York, USA.
Gold NB; Massachusetts General Hospital Department of Pediatrics, Boston, Massachusetts, USA.
Kennedy A; EveryLife Foundation for Rare Diseases, Washington, District of Columbia, USA.
Kishnani PS; Department of Pediatrics, Duke University, Durham, North Carolina, USA.
Might M; Hugh Kaul Precision Medicine Institute, University of Alabama at Birmingham, Birmingham, Alabama, USA.
Brooks PJ; Office of Rare Disease Research, National Center for Advancing Translational Science, National Institutes of Health, Bethesda, Maryland, USA.
Morris JA; Division of Neuroscience, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, Maryland, USA.
Parisi MA; Intellectual and Developmental Disabilities Branch, Eunice Kennedy Shriver, National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
Urv TK; Office of Rare Disease Research, National Center for Advancing Translational Science, National Institutes of Health, Bethesda, Maryland, USA.

Am J Med Genet C Semin Med Genet ; 193(1): 7-12, 2023 03.

Article em En | MEDLINE | ID: mdl-36691939

Assuntos

Terapia Genética; Doenças Raras; Humanos; Doenças Raras/genética; Doenças Raras/terapia

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Terapia Genética / Doenças Raras Limite: Humans Idioma: En Revista: Am J Med Genet C Semin Med Genet Assunto da revista: GENETICA MEDICA Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos

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