Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?

dc.creatorBraga, Luiza Amara Maciel
dc.creatorConte Filho, Carlos Gilbert
dc.creatorMota, Fabio Batista
dc.date2022-06-21T20:07:27Z
dc.date2022-06-21T20:07:27Z
dc.date2022
dc.date.accessioned2023-09-26T23:42:29Z
dc.date.available2023-09-26T23:42:29Z
dc.identifierBRAGA, Luiza Amara Maciel; CONTE FILHO, Carlos Gilbert; MOTA, Fabio Batista. Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?. Therapeutic Advances in Rare Disease, v. 3, p. 1 - 6, 2022.
dc.identifier2633-0040
dc.identifierhttps://www.arca.fiocruz.br/handle/icict/53407
dc.identifier10.1177/26330040221100840
dc.identifier.urihttps://repositorioslatinoamericanos.uchile.cl/handle/2250/8893384
dc.descriptionIntroduction: Rare genetic diseases affect millions of people worldwide. Most of them are caused by defective genes that impair quality of life and can lead to premature death. As genetic therapies aim to fix or replace defective genes, they are considered the most promising treatment for rare genetic diseases. Yet, as these therapies are still under development, it is still unclear whether they will be successful in treating these diseases. This study aims to address this gap by assessing researchers’ opinions on the future of genetic therapies for the treatment of rare genetic diseases. Methods: We conducted a global cross-sectional web-based survey of researchers who recently authored peer-reviewed articles related to rare genetic diseases. Results: We assessed the opinions of 1430 researchers with high and good knowledge about genetic therapies for the treatment of rare genetic diseases. Overall, the respondents believed that genetic therapies would be the standard of care for rare genetic diseases before 2036, leading to cures after this period. CRISPR-Cas9 was considered the most likely approach to fixing or replacing defective genes in the next 15 years. The respondents with good knowledge believed that genetic therapies would only have long-lasting effects after 2036, while those with high knowledge were divided on this issue. The respondents with good knowledge on the subject believed that non-viral vectors are more likely to be successful in fixing or replacing defective genes in the next 15 years, while most of the respondents with high knowledge believed viral vectors would be more successful. Conclusion: Overall, the researchers who participated in this study expect that in the future genetic therapies will greatly benefit the treatment of patients with rare genetic diseases.
dc.formatapplication/pdf
dc.languageeng
dc.publisherSage Publication
dc.rightsopen access
dc.subjectOpinião de um 'expert
dc.subjectFuturo
dc.subjectTerapias genéticas
dc.subjectDoenças genéticas raras
dc.subjectEnquete
dc.subjectExpert opinion
dc.subjectFuture
dc.subjectGenetic therapies
dc.subjectRare genetic diseases
dc.subjectSurvey
dc.titleFuture of genetic therapies for rare genetic diseases: what to expect for the next 15 years?
dc.typeArticle


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