Introduction: 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. Plain Language Summary A global survey of researchers on the future of genetic therapies for rare genetic diseases Rare genetic diseases are caused by defective genes that result from one or more mutations in the genome. Today, the therapeutic options for these diseases are limited, and there are approved treatments for about 5% of them. In the future, genetic therapies (a group of techniques developed to correct defective genes) are expected to revolutionize the treatment of rare genetic diseases. Although promising, most of these therapies are currently under development and have a long way to go before their efficacy and safety can be proved. The uncertainty surrounding this topic therefore means the success of genetic therapies in treating or curing rare genetic diseases is not yet assured. To address this knowledge gap, we surveyed 1430 researchers working in rare genetic diseases about the future of genetic therapies for the treatment of these diseases over the next 15 years. Most of them expected gene therapies to be the standard of care for rare genetic diseases before 2036 and to be able to cure them after this date. CRISPR-Cas9 was felt to be the gene editing approach that was most likely to succeed in fixing or replacing defective genes in the next 15 years. The respondents with high knowledge about gene therapies for the treatment of rare diseases believed gene therapies would have long-lasting effects before 2036, while those with good knowledge expected this to be the case only after 2036. The former believed in viral vectors and the latter in non-viral vectors to fix or replace defective genes in the next 15 years.
Purpose Lab on-a-chip (LOC) may lead to low-cost point-of-care devices for the diagnosis of human diseases, possibly making laboratories dispensable. However, as it is still an emerging technology, very little is known about its future impact on the diagnosis of human diseases, and on the laboratory industry. Hence, the purpose of this study is to foresee possible developments of this technology through a consultation with researchers in the field in two distinct time periods. Design/methodology/approach Based on Technology Foresight, this study addresses this gap by assessing the opinions of over five hundred LOC researchers and tracking changes in their views on the future of LOC diagnostic devices. These researchers participated in a two-wave global survey with an interval of two and a half years Findings Although second-wave (2020) respondents are less optimistic than those of the first wave (2017), the results of both surveys show that LOC diagnostic devices are expected to: move from proof-of-concept demonstrations to industrial development, becoming commercially feasible worldwide; integrate all laboratory processes, delivering cheaper, faster and more reliable diagnoses than laboratories; and provide low-cost point-of-care solutions, improving access to healthcare. Research limitations/implications Although it would be desirable to collect and explore the views of different sets of stakeholders, the method of generating lists of survey respondents shows a bias toward academic/scientific circles because the respondents are authors of scientific publications. These publications may as well be authored by stakeholders from other fields but it is reasonable to assume that most of them are researchers affiliated with universities and research and development organizations. Therefore, this study lacks in providing an image of the future based on a more diverse set of respondents. Social implications The results show that these devices are expected to radically change the diagnostic testing market and the way laboratories are organized, perhaps moving to a non-laboratory-based model. In conclusion, in the coming decades, these devices may promote substantial changes in the way human diseases are diagnosed. Originality/value Only a few studies have attempted to foresee the future of LOC devices, and most are based on literature reviews. Thus, this study goes beyond the existing research by providing a broad understanding of what the future will look like from the views of researchers who are contributing to the advancement of knowledge in the field. The researchers invited to take part in this study are authors of LOC-related scientific publications indexed in the Web of Science Core Collection.
Este trabalho objetivou analisar a influência (negativa ou positiva) de variáveis macro e microeconômicas no preço das ações da Petrobras, pelo período de 12 anos, entre o ano de 2009 e o ano de 2020. Os métodos empregados foram: i) correlação de Pearson e; ii) Mínimos Quadrados Ordinários (MQO). Os fatores de natureza qualitativa foram analisados com base numa revisão de literatura. Os principais resultados, obtidos a partir das metodologias empregadas, apontaram que a ação da Petrobras tem o seu preço afetado, principalmente, por fatores como: preço do petróleo Brent, índice Ibovespa e nível de Produção. Estas variáveis foram estatisticamente significantes, no MQO, para explicar as oscilações do preço da ação PETR4. Por fim, vale ressaltar, ademais, que os Indicadores financeiros da empresa, bem como os cenários e as interferências políticas, manifestas, por exemplo, na política de preço dos combustíveis ou em atos de corrupção (que se apresentam como falhas de governança) no âmbito da empresa, podem influenciar, consideravelmente, o comportamento do preço da PETR4.
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