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.
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.
ELSIcon2022 • Moderated Plenary Panel • June 3, 2022
This final panel discussion features keynote and plenary speakers Ruqaiijah Yearby, Teresa Blankmeyer-Burke, and Keith Wailoo, moderated by Sandra Soo-Jin Lee and Mildred Cho.
ELSIcon2022 • Pre-recorded Flash
Sickle cell disease (SCD), characterized by severe pain crisis and progressive organ damage as well as early death, is one of the most common inherited blood disorders in the world. However, since 90% of U.S. patients self-identify as Black and experience neglect as a result of structural racism, until recently, the condition has received very little research funding and minimal attention from the medical research community. Recent advances in human genetics have reinvigorated interest in the genetic underpinnings of SCD and the potential to “cure” it with novel genetic therapies.