The role of stem cells in cystic fibrosis disease modeling and drug discovery

Conese, Massimo; Beccia, Elisa; Carbone, Annalucia; Castellani, Stefano; Gioia, Sante Di; Corti, Fabiola; Angiolillo, Antonella; Colombo, Carla

doi:10.1080/21678707.2018.1549480

Cited by 1 publication

(1 citation statement)

References 93 publications

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“…Then, we generated a list of their Digital Object Identifiers (DOIs) and imported it into the reference management software Citavi 6.1, where these references were downloaded in a PDF format and fully read. After this process, we considered 47 [ 6 , 8 , 9 , 10 , 20 , 22 , 23 , 24 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 47 , 48 , 49 , 50 , 51 , 52 , 53 , 54 , 55 , 56 , 57 , 58 , 59 , 60 , 61 , 62 , 63 , 64 ] references appropriate to the study and used them in the literature review that allowed us to draft the survey questionnaire.…”

Section: Methodsmentioning

confidence: 99%

Anticipating New Treatments for Cystic Fibrosis: A Global Survey of Researchers

Cabral

Terlizzi

Laselva

et al. 2022

JCM

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Cystic fibrosis is a life-threatening disease that affects at least 100,000 people worldwide. It is caused by a defect in the cystic fibrosis transmembrane regulator (CFTR) gene and presently, 360 CFTR-causing mutations have been identified. Since the discovery of the CFTR gene, the expectation of developing treatments that can substantially increase the quality of life or even cure cystic fibrosis patients is growing. Yet, it is still uncertain today which developing treatments will be successful against cystic fibrosis. This study addresses this gap by assessing the opinions of over 524 cystic fibrosis researchers who participated in a global web-based survey. For most respondents, CFTR modulator therapies are the most likely to succeed in treating cystic fibrosis in the next 15 years, especially through the use of CFTR modulator combinations. Most respondents also believe that fixing or replacing the CFTR gene will lead to a cure for cystic fibrosis within 15 years, with CRISPR-Cas9 being the most likely genetic tool for this purpose.

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