CRISPR trial shows promise against deadly disease
Preliminary results from a landmark clinical trial suggest that CRISPR–Cas9 gene editing can be deployed directly into the body to treat disease.
The study is the first to show that the technique can be safe and effective if the CRISPR–Cas9 components (illustration pictured) — in this case targeting a protein that is made mainly in the liver — are infused into the bloodstream. In the trial, six people with a rare and fatal condition called transthyretin amyloidosis received a single treatment with the gene-editing therapy. All experienced a drop in the level of a misshapen protein associated with the disease. Those who received the higher of two doses tested saw levels of the protein, called TTR, decline by an average of 87% (J. D. Gillmore et al. N. Engl. J. Med. https://doi.org/gkxvcs; 2021).
Previous CRISPR–Cas9 clinical trials have suggested that the technique can be used in cells that have been removed from the body. But to be able to edit genes directly in the body would open the door to treating a wider range of diseases.
“It’s an important moment for the field,” says Daniel Anderson, a biomedical engineer at the Massachusetts Institute of Technology in Cambridge. “It’s a whole new era of medicine.”
Early success for live-parasite malaria vaccine
An experimental malaria vaccine that contains live parasites protected nearly all recipients from infection in a small clinical trial.
Participants in the study, which was published on 30 June (A. Mwakingwe-Omari et al. Nature 595, 289–302; 2021), were given a shot containing live Plasmodium falciparum, along with drugs to kill any parasites that reached the liver or blood, where they can cause malaria symptoms. Participants were then intentionally infected with malaria three months later to test the vaccine’s efficacy.
The vaccination protected 87.5% of participants who were infected with the strain of parasite that was used in the inoculation, and 77.8% of those who were infected with a different strain. This is a significant improvement on earlier efforts to use live parasites in a malaria vaccine; these did not perform as well against different strains.
The study also yielded important information about how immunity against malaria can be achieved, says Pedro Alonso, director of the World Health Organization’s Global Malaria Programme in Geneva, Switzerland. “It contributes considerably to the science of vaccines,” he says.
Pandemic made parents consider quitting academia
The stress of balancing work and home life during the COVID-19 pandemic has left many medical scientists with children questioning their future careers, according to a survey at a US university.
Last September, researchers at the University of Texas Southwestern Medical Center in Dallas sent a survey about work–life balance to the more than 3,000 members of academic staff in the university’s faculty of medicine. Around one-third responded.
The survey asked about two periods of time: one before the pandemic, from March 2019 to March 2020, and one more recent, from March to September 2020.
The proportion of respondents considering leaving the university rose by 9 percentage points to 23% between the pre- and post-pandemic time periods, with women and working parents particularly affected.
The study (S. A. Matulevicius et al. JAMA Netw. Open 4, e2113539; 2021) concluded that an increase in reported work–life stress since the start of the pandemic “may disproportionately decrease the long-term retention and promotion of junior and midcareer women faculty”.