Skip to main content
Premium Trial:

Request an Annual Quote

Sickle Cell Down for the Count?

By switching off the BCL11A gene, researchers at Harvard Medical School succeeded in eliminating sickle cell disease in mice, reports New Scientist's Charlie Harvey. The gene is responsible for switching the body's hemoglobin from the fetal form to the adult form, and since it's a mutant form of adult hemoglobin that causes the disease, knocking out BCL11A tricks the body into making fetal hemoglobin, and eliminates the condition, he adds. The researchers, who recently published their work in Science, knocked out BCL11A in mice that develop a sickle cell-like disease. "As adults, the mice produced over 20 times more fetal hemoglobin than normal and their blood contained almost no sickle-shaped cells," Harvey says. "Their spleen and kidneys — organs easily damaged by the effects of the disease — were almost completely healthy." In theory, gene therapy based on this research could be developed to treat the disease — specially-designed RNA could be injected into the bloodstream to silence BCL11A. But as that approach would be expensive and difficult to achieve on a large scale, the researchers say their long-term goal is to create a drug that can block the function of BCL11A that can be taken by a large population.

The Scan

US Booster Eligibility Decision

The US CDC director recommends that people at high risk of developing COVID-19 due to their jobs also be eligible for COVID-19 boosters, in addition to those 65 years old and older or with underlying medical conditions.

Arizona Bill Before Judge

The Arizona Daily Star reports that a judge is weighing whether a new Arizona law restricting abortion due to genetic conditions is a ban or a restriction.

Additional Genes

Wales is rolling out new genetic testing service for cancer patients, according to BBC News.

Science Papers Examine State of Human Genomic Research, Single-Cell Protein Quantification

In Science this week: a number of editorials and policy reports discuss advances in human genomic research, and more.