Skip to main content
Premium Trial:

Request an Annual Quote

Treatment So Far

Initial tests indicate that a CRISPR-based treatment for sickle cell disease may be working, NPR reports.

In August, Vertex Pharmaceuticals and CRISPR Therapeutics began a trial in which doctors are removing cells from patients' bone marrow, editing them to produce fetal hemoglobin, and re-introducing the now-altered cells back into the patients. The trial, which is to have 45 patients, started with one, Victoria Gray, as NPR reported at the time.

It now reports that Gray's cells are producing fetal hemoglobin, and more than the researchers estimated would be needed to treat her sickle cell disease. Additionally, Gray has not had any painful attacks associated with the condition, has not had to receive any blood transfusions, and has been able to go watch her son play sports, it adds. This, NPR's Rob Stein says, is "a first hint that this [treatment] might be working" and safe.

"It's amazing, you know, to have a chance at a different type of life," Gray tells NPR.

Gray's doctor, TriStar Centennial Medical Center's Haydar Frangoul, notes that she will continue to be monitored, but that this is a "huge achievement."

The Scan

Latent HIV Found in White Blood Cells of Individuals on Long-Term Treatments

Researchers in Nature Microbiology find HIV genetic material in monocyte white blood cells and in macrophages that differentiated from them in individuals on HIV-suppressive treatment.

Seagull Microbiome Altered by Microplastic Exposure

The overall diversity and the composition at gut microbiome sites appear to coincide with microplastic exposure and ingestion in two wild bird species, according to a new Nature Ecology and Evolution study.

Study Traces Bladder Cancer Risk Contributors in Organ Transplant Recipients

In eLife, genome and transcriptome sequencing reveal mutation signatures, recurrent somatic mutations, and risky virus sequences in bladder cancers occurring in transplant recipients.

Genes Linked to White-Tailed Jackrabbits' Winter Coat Color Change

Climate change, the researchers noted in Science, may lead to camouflage mismatch and increase predation of white-tailed jackrabbits.