Journal
AMERICAN JOURNAL OF OBSTETRICS AND GYNECOLOGY
Volume 225, Issue 6, Pages B9-B18Publisher
MOSBY-ELSEVIER
Keywords
CRISPR-Cas9; fetal gene therapy; fetal therapy; in utero CRISPR-Cas9 gene editing; in utero fetal gene therapy; in utero gene editing; in utero gene therapy; sickle cell disease
Categories
Ask authors/readers for more resources
Despite obstacles such as incomplete understanding of the fetal immune system, maternal immune responses to in utero gene therapy, risks of off-target effects from gene editing, gestational age constraints, and ethical questions surrounding fetal genetic intervention, none of these barriers appears insurmountable, and the journey to in utero gene therapy for sickle cell disease and other conditions should be well underway.
With the recent advances in gene editing with systems such as CRISPR-Cas9, precise genome editing in utero is on the horizon. Sickle cell disease is an excellent candidate for in utero fetal gene therapy, because the disease is monogenic, causes irreversible harm, and has life-limiting morbidity. Gene therapy has recently been proven to be effective in an adolescent patient. Several hurdles still impede the progress for fetal gene therapy in humans, including an incomplete understanding of the fetal immune system, unclear maternal immune responses to in utero gene therapy, risks of off-target effects from gene editing, gestational age constraints, and ethical questions surrounding fetal genetic intervention. However, none of these barriers appears insurmountable, and the journey to in utero gene therapy for sickle cell disease and other conditions should be well underway.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available