Functional Profiling of CFTR-Directed Therapeutics Using Pediatric Patient-Derived Nasal Epithelial Cell Models.

Park, Jeffrey KiHyun; Shrivastava, Anura; Zhang, Chengkang; Pollok, Brian A; Finkbeiner, Walter E; Gibb, Elizabeth R; Ly, Ngoc P; Illek, Beate

Park, Jeffrey KiHyun; Shrivastava, Anura; Zhang, Chengkang; Pollok, Brian A; Finkbeiner, Walter E; Gibb, Elizabeth R; Ly, Ngoc P; Illek, Beate.

Afiliação

Park JK; UCSF Benioff Children's Hospital Oakland, Children's Hospital Oakland Research Institute, Oakland, CA, United States.
Shrivastava A; Propagenix Inc., Gaithersburg, MD, United States.
Zhang C; Propagenix Inc., Gaithersburg, MD, United States.
Pollok BA; Propagenix Inc., Gaithersburg, MD, United States.
Finkbeiner WE; Department of Pathology, UCSF and Zuckerberg San Francisco General Hospital, San Francisco, CA, United States.
Gibb ER; Department of Pediatrics, UCSF Benioff Children's Hospital San Francisco, San Francisco, CA, United States.
Ly NP; Department of Pediatrics, UCSF Benioff Children's Hospital San Francisco, San Francisco, CA, United States.
Illek B; UCSF Benioff Children's Hospital Oakland, Children's Hospital Oakland Research Institute, Oakland, CA, United States.

Front Pediatr ; 8: 536, 2020.

Article em En | MEDLINE | ID: mdl-33014932

Palavras-chave

3(21kb) (c.54-5940_270+10250del21 kb; 406-1G->A (c.274-1G>A); CFTRdele2; Cystic fibrosis; F508del (c.1521_1523delCTT; R117H-7T (c.350G>A; R334W (c.1000C>T; c.850dupA (977insA; mutation-specific CFTR function analysis; p.Arg117His); p.Arg334Trp); p.Met284Asnfs); p.Phe508del); p.Ser18ArgfsX16)

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Texto completo: 1 Base de dados: MEDLINE Idioma: En Revista: Front Pediatr Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo

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PubMed Links

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Texto completo: 1 Base de dados: MEDLINE Idioma: En Revista: Front Pediatr Ano de publicação: 2020 Tipo de documento: Article País de afiliação: Estados Unidos