Enlarge / Richard Watkins,49, (in bed) is suffering from complications caused by Sickle cell disease. (credit: Washington Post/Getty Images )

Gene therapy has had a long and sometimes difficult history. Plenty of human genetic disorders can be traced to problems with a single gene, and that makes them a tempting target for correction. But someone died in a very early gene-therapy trial, which set the entire field back considerably. And, despite a far more cautious approach, the risks are still considerable, as two deaths during a trial occurred just this year .

But for researchers in the field, and those suffering from genetic diseases, this week provides some hope that the field's long-delayed promise might eventually be met. At a virtual scientific conference, a group presented the results of a large safety trial that saw 50 of 52 patients able to discontinue treatments for hemophilia. And a separate paper describes the use of CRISPR gene-editing and a blood stem cell transplant to successfully treat patients with sickle cell anemia or a related disorder.

Restoring clotting

The hemophilia trial was typical of most early efforts at gene therapy. In this case, the disease is caused by a defect in a single gene, so providing cells with a new copy will correct the problem. And, since the protein that's encoded by that gene circulates in the blood, you don't have to target a small and potentially difficult-to-access population of cells in order to correct things—targeting a new copy of the gene to any cells that can export proteins to the bloodstream will work.

Enlarge / If you can't socially distance, a face mask helps. (credit: Christopher Furlong / Getty Images )

Many countries that controlled their COVID-19 cases in the spring are now seeing rises in infections, raising the prospect that they'll face a second wave of cases, as many epidemiological models had predicted. But in the United States, the number of cases has never dropped to low levels. Instead, it varied between high levels of infection and very high peaks in cases. Why is everything so different in the states?

While there are plenty of possible reasons, a series of new studies essentially blame all the obvious ones: the United States ended social distancing rules too soon, never built up sufficient testing and contact-tracing capabilities, and hasn't adopted habits like mask use that might help substitute for its failures elsewhere. The fact that some of these studies used very different methods to arrive at similar conclusions suggests that those conclusions are likely to hold up as more studies come in.

Too soon

One of the studies, performed by a US-South African team, looked at the relaxation of social distancing rules in the US. Its authors created a list of restrictions for each state and the District of Columbia and tracked the number of COVID-19 deaths in each state for eight weeks prior to the rules being terminated. The number of deaths was used as a proxy for the total number of cases, as the erratic availability of tests made the true infection rate difficult to determine.

Enlarge / MOSCOW, RUSSIA - SEPTEMBER 4, 2020: Medical staff with newly delivered boxes containing COVID-19 vaccine in a cold room at No2 Outpatient Clinic in southern Moscow. (credit: Stanislav Krasilnikov / Getty Images )

Russia has been one of the countries hit hard by the COVID-19 pandemic. But its response to that has been a bit... unusual. As many other countries have, Russia worked to develop its own vaccine. But while that development was still in progress, it announced that it wasn't going to wait for detailed safety data , and instead roll the vaccine out to millions. Shortly afterwards, it became clear that the country was actually going to run a standard phase 3 clinical trial , albeit a large one, involving 40,000 people.

It was hard to judge whether any of this was reasonable, because few details of the vaccine itself were available. But that changed somewhat on Friday, as the people who developed the vaccine published the results of the initial clinical trials. And so far, it seems to be about as effective as some of the other ones that have been made it past initial trials.

Two viruses better than one?

As our earlier coverage mentioned, the vaccine is composed of two different engineered viruses. These contain the backbone of an innocuous virus, called an adenovirus, engineered to include the gene that encodes the major surface protein from the SARS-CoV-2 virus. This protein, called Spike, is what the coronavirus uses to latch on to and enter cells. The use of adenovirus allows the immune system to learn to recognize the Spike protein while the body only experiences a harmless adenovirus infection.

Enlarge / FDA Commissioner Stephen Hahn, speaking at the press conference in which he badly mangled statistics. (credit: Pete Marovich/Getty Image )

After several days of rumors with ever-growing hype, the Trump administration announced on Sunday that the Food and Drug Administration was granting an Emergency Use Authorization (EUA) for a COVID-19 treatment. The move was controversial from the start, with reports indicating that the EUA was opposed by a number of health experts, including Francis Collins and Anthony Fauci. The press conference didn't settle matters, with a growing chorus of scientists saying that the data presented in support of the EUA had been misrepresented .

On Monday night, FDA commissioner Stephen Hahn acknowledged that he had made a significant error in presenting the benefits of the treatment, and he followed that with an apology on Tuesday . But Hahn pushed back on indications that the approval of the treatment on the eve of the Republican National Convention was motivated by political pressure.

Wrong kind of risk

The treatment at issue involves taking the antibody-containing plasma from those who have recovered from a SARS-CoV-2 infection (convalescent plasma) and giving it to those currently suffering from COVID-19 symptoms. At Sunday's press conference, the principle justification for allowing this treatment under an EUA was a 35 percent drop in mortality for those receiving plasma in the first three days of treatment—specifically, Hahn said 35 of 100 people "would have been saved" by this treatment.

Enlarge / After her research career effectively ended, Dr. Judy Mikovits has re-emerged as an anti-vaccine activist. (credit: YouTube)

Back in 2011, we covered the strange story of biochemist Judy Mikovits, who co-authored a controversial (and subsequently retracted) paper in the journal Science and eventually lost her prestigious position with a research institution. Now Mikovits is back in the news, having spent the ensuing years reinventing herself as a staunch anti-vaccine crusader.

The COVID-19 pandemic has given her a new conspiracy to tout, this time targeting Dr. Anthony Fauci, director of the National Institute of Allergy and Infectious Diseases at NIH, who has become a prominent public spokesperson during the outbreak. Two interviews in particular have been spreading rapidly on social media, prompting YouTube and Facebook to remove both video clips for spreading medical misinformation during a global pandemic—a violation of their current policies

In 2007, Mikovits met Robert Silverman at a conference. Silverman had co-discovered a retrovirus known as XMRV, closely related to a known virus from mice. He told her he had found XMRV sequences in specimens from prostate cancer patients, although other labs, using different sets of patients, could find no evidence of a viral infection. Nonetheless, this prompted Mikovits to use the same tools to look for XMRV in samples from patients suffering from chronic fatigue syndrome (CFS)—a disorder some had claimed was purely psychosomatic.