Early Results Are Positive for Experimental CRISPR Therapies
Early Results Are Positive for Experimental CRISPR Therapies
Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.
Early Results Are Positive for Experimental CRISPR Therapies
Early Results Are Positive for Experimental CRISPR Therapies

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

Two clinical trial participants—one with β-thalassemia and one with sickle cell disease—appeared to benefit from the gene-editing treatments with minimal side effects, according to the companies.

sickle cell anemia
In Our Blood: A Profile of Stuart Orkin
In Our Blood: A Profile of Stuart Orkin
Anna Azvolinsky | Jul 15, 2019
By unraveling the molecular underpinnings of inherited blood disorders, the Boston Children’s Hospital researcher has provided the basis for therapies now being tested for beta-thalassemia and sickle cell disease.
Charting Crescents, 1910
Charting Crescents, 1910
Sukanya Charuchandra | Oct 1, 2018
James Herrick, a Chicago doctor, was the first to describe sickled red blood cells in a patient of African descent.
US Companies Launch CRISPR Clinical Trial
US Companies Launch CRISPR Clinical Trial
Catherine Offord | Sep 3, 2018
The Germany-based study will test an ex vivo genome-editing therapy for the inherited blood disorder β-thalassemia.
More Success Fixing Sickle Cell Gene with CRISPR
More Success Fixing Sickle Cell Gene with CRISPR
Kerry Grens | Nov 9, 2016
Researchers say they have sufficient in vitro and animal data to apply for human testing.
CRISPR Corrects Sickle Cell-Causing Gene in Human Cells
CRISPR Corrects Sickle Cell-Causing Gene in Human Cells
Kerry Grens | Oct 13, 2016
Once implanted in mice, the edited stem cells produced normal hemoglobin.
How Blood Cells Thwart Malaria
Tia Ghose | Nov 10, 2011
The sickle cell anemia mutation may protect against malaria by preventing the parasite from sending dangerous proteins to the red blood cell surface.