Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis
Cystic fibrosis (CF) is a autosomal recessive, multisystemic disease caused by different mutations in the CFTR gene encoding CF transmembrane conductance regulator. Although symptom management is important to avoid complications, the approval of CFTR modulator drugs in the clinic has demonstrated si...
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| Format: | Article |
| Language: | English |
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MDPI AG
2022-12-01
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| Series: | Children |
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| Online Access: | https://www.mdpi.com/2227-9067/10/1/4 |
| _version_ | 1797444399234285568 |
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| author | Jessica Conti Claudio Sorio Paola Melotti |
| author_facet | Jessica Conti Claudio Sorio Paola Melotti |
| author_sort | Jessica Conti |
| collection | DOAJ |
| description | Cystic fibrosis (CF) is a autosomal recessive, multisystemic disease caused by different mutations in the CFTR gene encoding CF transmembrane conductance regulator. Although symptom management is important to avoid complications, the approval of CFTR modulator drugs in the clinic has demonstrated significant improvements by targeting the primary molecular defect of CF and thereby preventing problems related to CFTR deficiency or dysfunction. CFTR modulator therapies have positively changed the patients’ quality of life, especially for those who start their use at the onset of the disease. Due to early diagnosis with the implementation of newborn screening programs and considerable progress in the treatment options, nowadays pediatric mortality was dramatically reduced. In any case, the main obstacle to treat CF is to predict the drug response of patients due to genetic complexity and heterogeneity. Advances in 3D culture systems have led to the extrapolation of disease modeling and individual drug response in vitro by producing mini organs called “organoids” easily obtained from nasal and rectal mucosa biopsies. In this review, we focus primarily on patient-derived intestinal organoids used as in vitro model for CF disease. Organoids combine high-validity of outcomes with a high throughput, thus enabling CF disease classification, drug development and treatment optimization in a personalized manner. |
| first_indexed | 2024-03-09T13:11:05Z |
| format | Article |
| id | doaj.art-e89d1d522bd24402aa0ef6bdb7fb3d22 |
| institution | Directory Open Access Journal |
| issn | 2227-9067 |
| language | English |
| last_indexed | 2024-03-09T13:11:05Z |
| publishDate | 2022-12-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Children |
| spelling | doaj.art-e89d1d522bd24402aa0ef6bdb7fb3d222023-11-30T21:42:18ZengMDPI AGChildren2227-90672022-12-01101410.3390/children10010004Organoid Technology and Its Role for Theratyping Applications in Cystic FibrosisJessica Conti0Claudio Sorio1Paola Melotti2Department of Medicine, Division of General Pathology, University of Verona, 37134 Verona, ItalyDepartment of Medicine, Division of General Pathology, University of Verona, 37134 Verona, ItalyCystic Fibrosis Centre, Azienda Ospedaliera Universitaria Integrata Verona, 37126 Verona, ItalyCystic fibrosis (CF) is a autosomal recessive, multisystemic disease caused by different mutations in the CFTR gene encoding CF transmembrane conductance regulator. Although symptom management is important to avoid complications, the approval of CFTR modulator drugs in the clinic has demonstrated significant improvements by targeting the primary molecular defect of CF and thereby preventing problems related to CFTR deficiency or dysfunction. CFTR modulator therapies have positively changed the patients’ quality of life, especially for those who start their use at the onset of the disease. Due to early diagnosis with the implementation of newborn screening programs and considerable progress in the treatment options, nowadays pediatric mortality was dramatically reduced. In any case, the main obstacle to treat CF is to predict the drug response of patients due to genetic complexity and heterogeneity. Advances in 3D culture systems have led to the extrapolation of disease modeling and individual drug response in vitro by producing mini organs called “organoids” easily obtained from nasal and rectal mucosa biopsies. In this review, we focus primarily on patient-derived intestinal organoids used as in vitro model for CF disease. Organoids combine high-validity of outcomes with a high throughput, thus enabling CF disease classification, drug development and treatment optimization in a personalized manner.https://www.mdpi.com/2227-9067/10/1/4CFTRcystic fibrosisorganoidstheratypingpersonalized medicine |
| spellingShingle | Jessica Conti Claudio Sorio Paola Melotti Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis Children CFTR cystic fibrosis organoids theratyping personalized medicine |
| title | Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis |
| title_full | Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis |
| title_fullStr | Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis |
| title_full_unstemmed | Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis |
| title_short | Organoid Technology and Its Role for Theratyping Applications in Cystic Fibrosis |
| title_sort | organoid technology and its role for theratyping applications in cystic fibrosis |
| topic | CFTR cystic fibrosis organoids theratyping personalized medicine |
| url | https://www.mdpi.com/2227-9067/10/1/4 |
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