Skip to main content
Premium Trial:

Request an Annual Quote

Promising, But More Needed

Early results from two clinical trials are raising hopes for CRISPR-Cas9-based therapy for β-thalassemia and sickle cell disease, Nature Biotechnology reports

Last month, Vertex Pharmaceuticals and CRISPR Therapeutics announced that they had successfully treated two patients, one with β-thalassemia and one sickle cell, with CTX001. CTX001 treatment involves obtaining hematopoietic stem cells from patients, then altering them with CRISPR–Cas9 to increase the production of fetal hemoglobin, and re-introducing the engineered cells back into the patients. According to NPR, Victoria Gray, the sickle cell patient, has been producing fetal hemoglobin at levels higher than what the researcher estimated would be needed to treat her condition. The companies add that the β-thalassemia patient was transfusion free after nine months.

These, Nature Biotech writes, are encouraging outcomes. But, it adds that some researchers want to see more data. For instance, the Children's Hospital of Philadelphia's Stefano Rivella tells it that it is unclear how many patients the firms have treated thus far and whether the observed rise in HbF levels is due only to the treatment. Additionally, Rivella says it's also not yet clear whether this treatment approach would be appropriate for all forms of the disease. 

The Scan

Enzyme Involved in Lipid Metabolism Linked to Mutational Signatures

In Nature Genetics, a Wellcome Sanger Institute-led team found that APOBEC1 may contribute to the development of the SBS2 and SBS13 mutational signatures in the small intestine.

Family Genetic Risk Score Linked to Diagnostic Trajectory in Psychiatric Disorders

Researchers in JAMA Psychiatry find ties between high or low family genetic risk scores and diagnostic stability or change in four major psychiatric disorders over time.

Study Questions Existence of Fetal Microbiome

A study appearing in Nature this week suggests that the reported fetal microbiome might be the result of sample contamination.

Fruit Fly Study Explores Gut Microbiome Effects on Circadian Rhythm

With gut microbiome and gene expression experiments, researchers in PNAS see signs that the microbiome contributes to circadian rhythm synchronicity and stability in fruit flies.