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ANTICIPATING NEW TREATMENTS FOR CYSTIC FIBROSIS: A GLOBAL SURVEY OF RESEARCHERS
Terapias moduladoras CFTR
Terapias genéticas
Pesquisa
Opinião de um Expert
Affilliation
Universidade Federal da Bahia. Departamento de Economia. Salvador, BA, Brasil.
Cystic Fibrosis Regional Reference Center, Department of Paediatric Medicine, Anna Meyer Children’s University. 50139 Florence, Italy.
Department of Medical and Surgical Sciences, University of Foggia. 71122 Foggia, Italy.
Universidade Federal de Santa Maria. Departamento de Economia. Santa Maria, RS, Brasil.
Fundação Oswaldo Cruz. Instituto Oswaldo Cruz. Laboratório de Comunicação Celular. Rio de Janeiro, RJ, Brasil.
Cystic Fibrosis Regional Reference Center, Department of Paediatric Medicine, Anna Meyer Children’s University. 50139 Florence, Italy.
Department of Medical and Surgical Sciences, University of Foggia. 71122 Foggia, Italy.
Universidade Federal de Santa Maria. Departamento de Economia. Santa Maria, RS, Brasil.
Fundação Oswaldo Cruz. Instituto Oswaldo Cruz. Laboratório de Comunicação Celular. Rio de Janeiro, RJ, Brasil.
Abstract
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.
Keywords in Portuguese
Fibrose císticaTerapias moduladoras CFTR
Terapias genéticas
Pesquisa
Opinião de um Expert
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