Dog Study Revives Concerns About Virus Used for Gene Therapy
Dog Study Revives Concerns About Virus Used for Gene Therapy
Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.
Dog Study Revives Concerns About Virus Used for Gene Therapy
Dog Study Revives Concerns About Virus Used for Gene Therapy

Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.

Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.

blood disorder
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.
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.