Doctor Column

Historic First Gene Therapy for Sickle Cell Anemia December 25, 2023 Joseph R. Anticaglia, MD Britain’s medicines regulators in November 2023 approved the gene editing technology, CRISPR-Cas9, for the treatment of genetic diseases, such as sickle cell disease and thalassemia. This is the first licensed treatment aimed at curing the above two diseases. Much credit belongs to 2020 Nobel Prize laureates Jennifer Doudna. and Emmanuelle Charpentier for their innovating, landmark gene editing work. A Yellow Light for Gene Surgeons and Gene Surgery April 25, 2021 Joseph R. Anticaglia, MD Every year millions of infants (6% of worldwide births) are born with serious birth defects. In the United States, birth defects are the leading cause of infant mortality accounting for one in every 5 infant deaths. They afflict about three per cent of the babies born in the US. A 21st Century Scientific Revolution? CRISPR-Case 9 April 5, 2021 Joseph R. Anticaglia, MD Jennifer Doudna and Emmanuelle Charpentier have ignited a scientific revolution. In 2020, both shared the Nobel Prize in chemistry for their pioneering work in gene editing (also called genome editing or genetic engineering). This innovative technology, CRISPR-Cas9, allows scientists to more easily and accurately repair or enhance an organism’s faulty DNA.