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.
-
Jun 3, 2026, 15:54Karen Phillips: Surprisingly High Cumulative Clinical Bleeding Events In Post-Ablation AF Patients
-
Jun 3, 2026, 15:47Michael Makris: How Haemophilia Care Is Delivered in China
-
Jun 3, 2026, 15:36Alexandros Makis: 20-year Cohort of 112 Children with Autoimmune Neutropenia
-
Jun 3, 2026, 15:30Aravind Palraj: Young Stroke? – Think Beyond APS
-
Jun 3, 2026, 15:09Alissa Rumin: Starting a New Chapter in Hemophilia and Thrombosis Research
-
Jun 3, 2026, 15:02Yauheniya Baranchykava: Changing Lives Through Innovation in Hemophilia Care
-
Jun 3, 2026, 15:01Danielle Leitner Baxter: 15 Years of Supporting the Bleeding Disorders Community
-
Jun 3, 2026, 14:44Brandy Seyller: Why Understanding the Coagulation Cascade Matters in Bleeding Cases
-
Jun 3, 2026, 14:33Anna Tobiasch: Should Fibrinogen and FXIII Be Restored Together After Severe Haemodilution?