In Our Blood: A Profile of Stuart Orkin
In Our Blood: A Profile of Stuart Orkin
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.
In Our Blood: A Profile of Stuart Orkin
In Our Blood: A Profile of Stuart Orkin

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.

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.

blood disorder
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.
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 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.
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.