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Last Posted: Nov 23, 2022
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Africa must participate in finding a gene therapy cure for sickle-cell disease.
Moshi Grace et al. Nature medicine 2022 10

SCD is a disease of global public heath significance, has a dismal effect on the quality of life of many people, and it is a major drain on human and health resources. Scientific collaboration is needed to prioritize treatments that are cost-effective in Africa, where most cases exist. For these interventions to be implemented in Africa, several barriers must be overcome. Barriers include under-funded healthcare systems, lack of appropriate infrastructure, and lack of manufacturing practice facilities.

Sickle Cell Disease and Gene Therapy — Patient and Physician Perspectives
NEJM, Youtube video, Septenber 2022

In this short documentary video from the New England Journal of Medicine (NEJM), patients and physicians partner both to highlight the experience of living with sickle cell disease and to discuss the pathophysiology of the disease and new treatment strategies, including gene therapy.

Sickle Cell Disease and Gene Therapy - Patient and Physician Perspectives.
DeBaun Michael et al. The New England journal of medicine 2022 9 (13) e28

In this short documentary video, patients and physicians partner both to highlight the experience of living with sickle cell disease and to discuss the pathophysiology of the disease and new treatment strategies, including gene therapy. Patients share their own stories of interactions with the health care system and explore the challenging topics of racial disparity and health equity. Physicians express a cautious optimism as they review the risks and benefits of gene therapy.

Monoclonal antibodies for malaria prevention.
Aleshnick Maya et al. Molecular therapy : the journal of the American Society of Gene Therapy 2022 4 (5) 1810-1821

Despite several approaches to prevent established infection, no single approach offers complete protection, so a multilayered approach is needed. One potential layer, the monoclonal antibody. opens in new tab, is the focus of a new trial. The authors describe protection conferred by a long-acting, next-generation monoclonal antibody against controlled human malaria infection in healthy persons.


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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