Skip to main content
Premium Trial:

Request an Annual Quote

To Turn on the Other Hemoglobin

Doctors in the US have used the CRISPR gene-editing tool to try to treat a woman with sickle cell disease as part of a clinical trial, Popular Science reports.

Victoria Gray is taking part in a study being conducted by Vertex Pharmaceuticals and CRISPR Therapeutics, NPR adds. For it, doctors are removing cells from patients' bone marrow and editing those cells to produce fetal hemoglobin, before then re-introducing the cells back in to the patients. People with sickle cell disease have faulty hemoglobin, but by editing the repressor of fetal hemoglobin — which is typically only expressed very early in life — the researchers hope to spur patients to produce that version of the protein to then ease their disease, Popular Science adds.

In all, the companies plan to enroll about 45 patients in their study, NPR says.

While NPR notes that it will be some time before doctors can tell if the treatment is working, Gray tells it she is envisioning a life without sickle cell disease. "Being able to wake up and not be in pain," she says. "And to just be tired because I've done something — and not just tired for no reason. And just to be outside, and jump on the trampoline with my kids."

The Scan

Positive Framing of Genetic Studies Can Spark Mistrust Among Underrepresented Groups

Researchers in Human Genetics and Genomics Advances report that how researchers describe genomic studies may alienate potential participants.

Small Study of Gene Editing to Treat Sickle Cell Disease

In a Novartis-sponsored study in the New England Journal of Medicine, researchers found that a CRISPR-Cas9-based treatment targeting promoters of genes encoding fetal hemoglobin could reduce disease symptoms.

Gut Microbiome Changes Appear in Infants Before They Develop Eczema, Study Finds

Researchers report in mSystems that infants experienced an enrichment in Clostridium sensu stricto 1 and Finegoldia and a depletion of Bacteroides before developing eczema.

Acute Myeloid Leukemia Treatment Specificity Enhanced With Stem Cell Editing

A study in Nature suggests epitope editing in donor stem cells prior to bone marrow transplants can stave off toxicity when targeting acute myeloid leukemia with immunotherapy.