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Last Posted: May 17, 2023
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Emerging Trends in Gene Therapy: Thalassemia as a Case Study
CDC Seminar— June 22, 2023, 2:00–3:00 PM ET Brand

Although relatively new in terms of clinical application, several gene therapy-based treatments have, in recent years, received approval from the Food and Drug Administration (FDA) and begun to be used in real world settings in the United States. In addition, clinical trials using either gene transfer or genome editing continue to show promise, with the potential to impact treatment for patients with a wide range of hereditary disorders in the future. This webinar will showcase thalassemia as a case example in emerging approaches in gene therapy.

Human embryo science: can the world’s regulators keep pace? Concerned by the speed of research, policymakers are looking for alternative ways to establish rules on genome editing and other technologies.
P Ball, Nature, May 10, 2023

The World Health Organization currently recommends against the use of heritable (or germline) human genome editing, but has no powers of enforcement — and neither does any other international body. Some national bodies, such as the HFEA, can create rules to govern research and its outputs, but such centralized oversight is relatively rare. Most scientists comply with guidelines formulated by professional bodies such as the International Society for Stem Cell Research (ISSCR), but these principles have neither legal force nor a mandate from wider society.

The ethics, equity, and governance of human genome editing need greater consideration.
Sarojini Nadimpally et al. BMJ 2023 5 p996

Challenges exist around heritable gene editing, its potential medical applications, ethical implications, and the need for regulatory mechanisms in the field. Adopting a purely scientific view of human genome editing risks ignoring ethical, societal, and equity considerations.

In the business of base editors: Evolution from bench to bedside.
Elizabeth M Porto et al. PLoS Biol 2023 4 (4) e3002071

With the advent of recombinant DNA technology in the 1970s, the idea of using gene therapies to treat human genetic diseases captured the interest and imagination of scientists around the world. Years later, enabled largely by the development of CRISPR-based genome editing tools, the field has exploded, with academic labs, startup biotechnology companies, and large pharmaceutical corporations working in concert to develop life-changing therapeutics.

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Disclaimer: Articles listed in the Public Health Genomics and Precision Health Knowledge Base are selected by the CDC Office of Public Health Genomics to provide current awareness of the literature and news. Inclusion in the update does not necessarily represent the views of the Centers for Disease Control and Prevention nor does it imply endorsement of the article's methods or findings. CDC and DHHS assume no responsibility for the factual accuracy of the items presented. The selection, omission, or content of items does not imply any endorsement or other position taken by CDC or DHHS. Opinion, findings and conclusions expressed by the original authors of items included in the update, or persons quoted therein, are strictly their own and are in no way meant to represent the opinion or views of CDC or DHHS. References to publications, news sources, and non-CDC Websites are provided solely for informational purposes and do not imply endorsement by CDC or DHHS.

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