Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype-Reference-Cited by-同舟云学术

Ex vivo model predicted in vivo efficacy of CFTR modulator therapy in a child with rare genotype

Author:

Terlizzi Vito¹^ORCID,Amato Felice²³,Castellani Chiara¹,Ferrari Beatrice¹,Galietta Luis J. V.⁴⁵,Castaldo Giuseppe²³,Taccetti Giovanni¹

Affiliation:

1. Cystic Fibrosis Regional Reference Center, Department of Paediatric Medicine Anna Meyer Children's University Florence Italy

2. Department of Molecular Medicine and Medical Biotechnology University of Naples Federico II

3. CEINGE – Advanced Biotechnologies Naples Italy

4. Telethon Institute of Genetics and Medicine (TIGEM Pozzuoli Italy)

5. Department of Translational Medical Sciences University of Naples Federico II Napoli Italy

Funder

Ministero della Salute

Publisher

Wiley

Subject

Genetics (clinical),Genetics,Molecular Biology

Link

Reference29 articles.

5. Rectal organoids enable personalized treatment of cystic fibrosis;Berkers G.;Cell Reports,2019

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