Ronan Kelly, Director of Precision Medicine and Scientific Discovery at Baylor University Medical Center, shared a post on LinkedIn:
“We are entering a new era of precision medicine!
Congratulations to Dr Luis Pineiro who leads our Bone Marrow Transplant program at Baylor University Medical Center in Dallas and his Ruby trial colleagues on today’s publication in the New England Journal of Medicine.
Gene editing therapies for sickle cell disease (SCD) continue to evolve. The data from this phase 1-2 study demonstrates an ability to address the underlying genetic cause of disease – rather than just managing the symptoms.
This represents hope that a brighter future may be ahead for all those suffering from SCD and other genetic disorders.”
Title: CRISPR-Cas12a Gene Editing of HBG1 and HBG2Promoters to Treat Sickle Cell Disease
Authors: Rabi Hanna, Haydar Frangoul, Luis Pineiro, Christopher McKinney, Markus Mapara, Jignesh Dalal, Hemalatha G Rangarajan, Harold Atkins, Akshay Sharma, Kai-Hsin Chang, Michael C Jaskolka, Keunpyo Kim, Qifeng Yu, Baisong Mei, Olubunmi Afonja, Mark C Walters, RUBY Investigators
Read the Full Article.
Other articles featuring Ronan Kelly.