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.

thalassemia
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.
In Utero Transplant in First Clinical Trial Successful
In Utero Transplant in First Clinical Trial Successful
Jim Daley | May 30, 2018
Doctors treated the fetus, who has alpha thalassemia major, with cells from her mother’s bone marrow.
Technique Adapted from CRISPR-Cas9 Corrects Mutation in Human Embryos
Technique Adapted from CRISPR-Cas9 Corrects Mutation in Human Embryos
Catherine Offord | Sep 28, 2017
Researchers use base-editing to swap out an erroneous nucleotide responsible for a potentially life-threatening blood disorder.
CRISPR Corrects Blood Disorder Gene
CRISPR Corrects Blood Disorder Gene
Kerry Grens | Aug 5, 2014
Scientists use the genome-editing technique to fix a disease-causing mutation in human cell lines.