Sickle Cell Disease (SCD) is an inherited disorder that results in premature mortality and severe morbidity in millions of afflicted individuals worldwide. Novel therapies are urgently needed and research to improve therapies for SCD is an NHLBI priority. Clinical trials using new methods of gene manipulation have begun in adults; however, it is most desirable to treat children, adolescents and young adults (AYA) before the onset of irreversible organ damage.
About this listing
ELSI in Review is a listing of recently published reviews of the literature on key ELSI topics curated by CERA staff. Our April 2025 set explores ethical considerations of prenatal gene editing for neurodevelopmental diseases, factors that influence parents’ decisions regarding pediatric genetic testing, and more. If you would like your ELSI-relevant review featured in this communication, please contact us at [email protected].
About this listing
ELSI in Review is a listing of recently published reviews of the literature on key ELSI topics curated by CERA staff. Our January 2025 set explores public perceptions of genetic engineering, ethical issues in healthcare artificial intelligence, and more. If you would like your ELSI-relevant review featured in this communication, please contact us at [email protected].
After 44 days, Kendric Cromer, 12, left the hospital. While his family feels fortunate that he was the first to receive a treatment, their difficult experiences hint at what others will be up against.
This course provides an introduction to key issues relating to genomics in society. It explores social, ethical and policy questions raised by genomics in the context of: synthetic biology, genetics and crops, genetic modification of domestic animals, genetics and conservation, human genetic therapies and human enhancement, and privacy and genetic information. The course was developed by Claire Palmer, Penny Riggs, T.J. Kasperbauer, Jeremy Johnson, Lauren Cifuentes, Seung Won Park, and Jamie McQueen.
Regulators in the U.K. on Thursday approved a CRISPR-based medicine to treat both sickle cell disease and beta thalassemia, making it the world’s first therapy built on the revolutionary gene-editing technology and ushering in a new era of genetic medicine.
The decision by an advisory committee may lead to Food and Drug Administration approval of the first treatment for humans that uses the CRISPR gene-editing system
Rare diseases, most of which have a genetic basis, collectively affect an estimated 300 million individuals worldwide and are associated with significant morbidity and mortality. Although less than 10% of rare diseases currently have an approved therapy, advances in gene and gene-targeted therapies have brought hope of effective—or even curative—treatment. While the potential for cure has prompted justifiable excitement, the high upfront cost of many of these therapies also raises complex ethical and policy issues.
ELSIcon2022 • Plenary Session • June 2, 2022
Welcome and Introduction by Sandra Soo-Jin Lee, PhD.
Plenary: "Gene Editing and Gene Therapy: The Case of Sickle Cell " presented by Keith Wailoo, PhD.