Article
Future of genetic therapies for rare genetic diseases: what to expect for the next 15 years?
Registro en:
BRAGA, 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.
2633-0040
10.1177/26330040221100840
Autor
Braga, Luiza Amara Maciel
Conte Filho, Carlos Gilbert
Mota, Fabio Batista
Resumen
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.