CRISPR-Based Gene Therapies Advance for Sickle Cell Disease and Beta-Thalassemia, Raising Ethical Questions on Germline Editing
CRISPR-based gene therapies are increasingly being used in clinical settings for somatic conditions such as sickle cell disease and beta-thalassemia. The U.S. FDA approved Casgevy, the world's first CRISPR-based gene therapy, in December 2023 for sickle cell disease. This therapy involves editing somatic cells, meaning the modifications affect only the treated individual and are not heritable. However, the rapid advancement of gene-editing technology, particularly CRISPR-Cas9, has also brought germline editing closer to reality. Germline editing, which involves modifying sperm, eggs, or early-stage embryos, results in heritable changes that are passed down to future generations. This distinction is critical, as the person most affected by a germline edit does not yet exist at the time of the intervention, raising significant ethical and legal concerns regarding consent and long-term consequences. The controversial 2018 case of Chinese biophysicist He Jiankui, who edited the germline of human embryos, spark...
