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
Catherine Offord | Sep 3, 2018 | 2 min read
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
Jim Daley | May 30, 2018 | 2 min read
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
Catherine Offord | Sep 28, 2017 | 2 min read
Researchers use base-editing to swap out an erroneous nucleotide responsible for a potentially life-threatening blood disorder.
CRISPR Corrects Blood Disorder Gene
Kerry Grens | Aug 5, 2014 | 2 min read
Scientists use the genome-editing technique to fix a disease-causing mutation in human cell lines.