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Eric Topol: Latest Clinical Trials of Genome Editing for Sickle Cell Disease and β-Thalassemia
Eric Topol/X
Apr 5, 2026, 15:26

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

Eric Topol: Latest Clinical Trials of Genome Editing for Sickle Cell Disease and β-Thalassemia

 

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

Eric Topol: Latest Clinical Trials of Genome Editing for Sickle Cell Disease and β-Thalassemia

 

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

Eric Topol: Latest Clinical Trials of Genome Editing for Sickle Cell Disease and β-Thalassemia

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

Eric Topol: Latest Clinical Trials of Genome Editing for Sickle Cell Disease and β-Thalassemia

Stay updated on all scientific advances with Hemostasis Today.

 

Akshay Sharma Alex C. Minella Alexis A. Thompson Alexis Thompson Amy Simon Ashish O. Gupta Asif Alavi Bahru Habtemariam Baisong Mei Christopher McKinney Clinton Carroll Edward D. Ziga Ernesto Ayala Harold Atkins Haydar Frangoul Health Hemalatha G. Rangarajan Hematology Hemophilia Hemostasis News Hemostasis Today Jennifer J. Jaroscak Jignesh Dalal John F. DiPersio Julie Kanter Kai-Hsin Chang Keunpyo Kim Ling Lin Luis Pineiro Marcelyne Joseney-Antoine Mark C. Walters Markus Mapara Markus Y. Mapara Mary Eapen Matthew M. Heeney Medicine Meghann McManus Michael C. Jaskolka Nnenna Badamosi Olubunmi Afonja Priya S. Chockalingam Qifeng Yu Rabi Hanna Roy L. Kao Sarah Chesler Srikanth Ambati Stacey Rifkin-Zenenberg Sunita Goyal Thomas S. Bowman Yinzhong Chen
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