Eric Topol: Latest Clinical Trials of Genome Editing for Sickle Cell Disease and β-Thalassemia
Eric Topol, Founder and Director of Scripps Research Translational Institute, shared a post on X about recent articles, published in NEJM:
“3 new trial results for genome editing of sickle cell disease and β-thalassemia.”
Title: More Options for Gene Editing in Hemoglobinopathies
Author: Franco Locatelli
Read the Full Article on NEJM
Title: Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease
Aurthors: Ashish O. Gupta, Akshay Sharma, Haydar Frangoul, Julie Kanter, Markus Y. Mapara, Jignesh Dalal, Asif Alavi, Jennifer J. Jaroscak, Ernesto Ayala, John F. DiPersio, Edward D. Ziga, Mary Eapen, Stacey Rifkin-Zenenberg, Alex C. Minella, Yinzhong Chen, Sarah Chesler, Srikanth Ambati, Thomas S. Bowman, Bahru Habtemariam, Marcelyne Joseney-Antoine, Priya S. Chockalingam, Ling Lin, Sunita Goyal, Amy Simon, Alexis A. Thompson, Matthew M. Heeney
Read the Full Article on NEJM
Title: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters 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
Read the Full Article on NEJM
Title: CRISPR-Cas12a Gene Editing of HBG1 and HBG2 Promoters to Treat β-Thalassemia
Aurhors: : Haydar Frangoul, Rabi Hanna, Mark C. Walters, Roy L. Kao, Clinton Carroll, Meghann McManus, Kai-Hsin Chang, Michael C. Jaskolka, Keunpyo Kim, Qifeng Yu, Nnenna Badamosi, Baisong Mei, Olubunmi Afonja, Alexis Thompson
Read the Full Article on NEJM
Stay updated on all scientific advances with Hemostasis Today.
-
Apr 11, 2026, 13:51David McIntosh: Vital Plasma Derived Medicines – The Anomalous UK Scene
-
Apr 11, 2026, 13:47Samrawit Terefe: O Negative Blood Is the Universal Donor With Extreme Scarcity
-
Apr 11, 2026, 13:39Dheeraj Garg: Rethinking Cardiovascular Disease – A Cardiologist’s Perspective
-
Apr 11, 2026, 13:37Kushal Bhatia: Is The 4.5-Hour Thrombolysis Window Officially Outdated?
-
Apr 11, 2026, 13:35William Aird: Why Did Mammalian Red Blood Cells Give Up Their Nucleus?
-
Apr 11, 2026, 13:30Ken Kuang: Gravity Is Constant, But Your Vein Health Doesn’t Have to Be
-
Apr 11, 2026, 13:21Mascha Kern: Measuring Gender’s Role in Stroke and Migraine
-
Apr 11, 2026, 13:10Danique Steeghs: Key Findings from a Microfluidic Chemiluminescent Thrombin Generation Assay
-
Apr 11, 2026, 13:06Shanvi Mahi: A Validated Tool to Explore Lived Experiences After Stroke Rehabilitation