A decade after gene therapy

Evangelina Vaccaro, above, who received the gene therapy
 for ADA-SCID in a clinical trial in 2012.
Credit: Courtesy of Alysia Padilla-Vaccaro

Over a decade ago, UCLA physician-scientists began using a pioneering gene therapy they developed to treat children born with a rare and deadly immune system disorder. They now report that the effects of the therapy appear to be long-lasting, with 90% of patients who received the treatment eight to 11 years ago still disease-free.

ADA-SCID, or adenosine deaminase–deficient severe combined immunodeficiency, is caused by mutations in the gene that creates the ADA enzyme, which is essential to a functioning immune system. For babies with the disease, exposure to everyday germs can be fatal, and if untreated, most will die within the first two years of life.

In the gene therapy approach detailed in the new paper, Dr. Donald Kohn of UCLA and his colleagues removed blood-forming stem cells from each child’s bone marrow, then used a specially modified virus, originally isolated from mice, to guide healthy copies of the ADA gene into the stem cells’ DNA. Finally, they transplanted the cells back into the children’s bone marrow. The therapy, when successful, prompts the body to produce a continuous supply of healthy immune cells capable of fighting infections. Because the transplanted stem cells are the baby’s own, there is no risk of rejection.

New technique, effective in mice, could help advance the use of probiotics

Quanyin Hu

Scientists studying probiotics, beneficial bacteria that show promise for their ability to treat inflammatory bowel disease and other intestinal disorders, continue to face a problem: how to keep probiotics from getting obliterated in the gut before they can be helpful.

Researchers at the University of Wisconsin–Madison School of Pharmacy think they have a solution involving a dual-layer protection system that can keep probiotic bacteria alive in the lower intestine long enough to help treat or prevent colitis in a mouse model of the disease.

Quanyin Hu and postdoctoral researcher Jun Liu have filed for a patent on their double-protection technique and, with their collaborators, published their findings in the journal Nano Today.

If the new technique shows similar effectiveness in clinical trials, it could help advance the use of probiotics.

The research addresses one of the biggest limitations of treating disease with probiotics, which are bacteria that promote healthy tissue development and improve the gut microbiome.

“When you transfer these bacteria through the oral route, most of them are getting killed by the acidic environment of the stomach. Or they’re getting cleared out of the intestine because they aren’t adhering,” says Hu, the senior author of the report. “Our double protection technique addresses these limitations.”

Gel fights drug-resistant bacteria

In the fight against multidrug-resistant bacteria, scientists in Sweden have developed a new kind of antibiotic-free protection for wounds that kills drug-resistant bacteria and induces the body’s own immune responses to fight infections.

Reporting in the Journal of the American Chemical Society, researchers from KTH Royal Institute of Technology, Karolinska Institutet and Karolinska University Hospital say that the new treatment is based on specially-developed hydrogels consisting of polymers known as dendritic macromolecules.

KTH Professor Michael Malkoch says the hydrogels are formed spontaneously when sprayed on wounds and 100 percent degradable and non-toxic.

“Dendritic hydrogels are excellent for wound dressing materials because of their soft, adhesive and pliable tactile properties, which provide ideal contact on the skin and maintain the moist environment beneficial for optimal wound healing,” he says.

Before and after. On the left, bacteria prior to exposure to the dendritic hydrogel, and on the right, the bacteria is visibly damaged.

The antibacterial effects of the hydrogels have yet to be fully understood, but the key lies in these macromolecules’ structure. It’s distinguished by well-ordered branches that terminate with a profusion of cationic, charged contact points.

How T cell assassins reload their weapons to kill and kill again

Cytotoxic T cells are specialist white blood cells that are trained by our immune system to recognize and eliminate threats – including tumor cells and cells infected with invading viruses, such as SARS-CoV-2, which causes COVID-19. They are also at the heart of new immunotherapies that promise to transform cancer treatment.

Professor Gillian Griffiths from the Cambridge Institute for Medical Research, who led the research, said: “T cells are trained assassins that are sent on their deadly missions by the immune system. There are billions of them in our blood, each engaged in a ferocious and unrelenting battle to keep us healthy.

“Once a T cell has found its target, it binds to it and releases its toxic cargo. But what is particularly remarkable is that they are then able to go on to kill and kill again. Only now, thanks to state-of-the-art technologies, have we been able to find out how they reload their weapons.”

Covid Lockdown: children who spent more time in nature fared best

Credit: Ben Wicks on Unsplash

A study has found that children who increased their connection to nature during the first COVID-19 lockdown were likely to have lower levels of behavioral and emotional problems, compared to those whose connection to nature stayed the same or decreased - regardless of their socio-economic status.

The study, by researchers at the University of Cambridge and the University of Sussex, also found that children from affluent families tended to have increased their connection to nature during the pandemic more than their less affluent peers.

Nearly two thirds of parents reported a change in their child’s connection to nature during lockdown, while a third of children whose connection to nature decreased displayed increased problems of wellbeing - either through ‘acting out’ or by increased sadness or anxiety.

The results strengthen the case for nature as a low-cost method of mental health support for children, and suggest that more effort should be made to support children in connecting with nature - both at home and at school.

The researchers’ suggestions for achieving this include: reducing the number of structured extracurricular activities for children to allow for more time outside, provision of gardening projects in schools, and funding for schools, particularly in disadvantaged areas, to implement nature-based learning programs.

Pesticide linked to chronic kidney disease

A commonly available pesticide has been associated with an increased risk of chronic kidney disease (CKD) in a University of Queensland study.

Researchers analyzed links between pesticide exposure and the risk of kidney dysfunction in 41,847 people, using data from the USA National Health and Nutrition Examination Survey (NHANES).

School of Public Health Associate Professor, Nicholas Osborne said the study found people exposed to higher amounts of the insecticide Malathion, known as Maldison in Australia, had 25 per cent higher risk of kidney dysfunction.

“Nearly one in 10 people in high income countries show signs of CKD, which is permanent kidney damage and loss of renal function,” Dr Osborne said.

Risk factors of developing CKD include age, hypertension and diabetes.

Dr Osborne said CKD with no known cause was rising in low-to-middle income countries such as India, Sri Lanka and Mesoamerica.

“Initially, it was suspected the condition was associated with agricultural workplaces through exposure to heat stress, dehydration, pesticide spraying, heavy metals and agrochemicals,” Dr Osborne said.

Identifying Better Ways to Save the World’s Reefs

Scientists work to restore damaged coral reefs by
growing health fragments to transplant.
Photo: University of Konstanz/Anna Rolk.

In the race to save the world’s fragile corals from climate change and disease, USF Department of Integrative Biology Assistant Professor John Parkinson is among an international group of scientists looking for better ways to restore damaged reefs. In new research published Tuesday in the journal Nature Reviews Earth & Environment, the team makes its case for nature-based approaches that give coral colonies a boost in their fight to survive warming seas.

Led by the University of Konstanz in Germany, the team calls for the use of new technologies to identify, propagate, and even strengthen naturally resilient coral colonies, which can then be transplanted to threatened reefs. The systematic review evaluates several emerging methods that have been used to improve coral heat tolerance, scoring them in terms of risk, cost, and scalability. The objective of the project is to provide guidance on which interventions might most effectively extend the natural adaptive capacity of corals, the researchers said.

The race to save the world’s coral reefs is a high priority for conservationists: 30 percent of all marine biodiversity depends on reef ecosystems and more than one billion people rely on the reefs for sustaining fishing stocks, tourism, and a healthy marine environment. Even a small rise in ocean temperatures can cause coral “bleaching” – when corals expel the algae living in their tissues causing the coral to turn completely white – leaving the organisms more susceptible to disease and death.

Flu season is coming and it could be ugly

Photo by Usman Yousaf on Unsplash

As COVID-19 restrictions ease, Canada is seeing a resurgence of many respiratory viruses—and many experts predict this year’s flu season could be severe.

Dr. Michael Curry (he/him), clinical associate professor with UBC faculty of medicine’s department of emergency medicine, reveals how this year’s flu season will be different from the last and weighs in on how to reduce your risk of getting sick.

How will this year’s flu season be different from last year?

Last flu season we essentially had no cases of influenza and relatively few cases of other respiratory viruses here in Canada. When someone was sick with a respiratory infection eight or nine months ago, it was very likely they had COVID-19.

This year, the big change I have seen is the re-emergence of our ‘usual suspect’ respiratory infections. Common cold viruses are back and circulating again. While we are still seeing COVID-19 in Canada, we are finding other respiratory viruses on a regular basis as well.

How might this year’s flu season interact with the fourth wave of COVID-19?

With the upsurge in other respiratory viruses, we can expect a resurgence of influenza this year.

A bad flu season can rapidly fill up emergency departments and hospital beds, and as we all know, COVID-19 is already doing a good job at that.

How many people get 'long COVID?'

Photo by Polina Tankilevitch from Pexels

More than half of the 236 million people who have been diagnosed with COVID-19 worldwide since December 2019 will experience post-COVID symptoms — more commonly known as “long COVID” — up to six months after recovering, according to Penn State College of Medicine researchers. The research team said that governments, health care organizations and public health professionals should prepare for the large number of COVID-19 survivors who will need care for a variety of psychological and physical symptoms.

During their illnesses, many patients with COVID-19 experience symptoms, such as tiredness, difficulty breathing, chest pain, sore joints and loss of taste or smell.

Until recently, few studies have evaluated patients’ health after recovering from the coronavirus. To better understand the short- and long-term health effects of the virus, the researchers examined worldwide studies involving unvaccinated patients who recovered from COVID-19. According to the findings, adults, as well as children, can experience several adverse health issues for six months or longer after recovering from COVID-19.

The researchers conducted a systematic review of 57 reports that included data from 250,351 unvaccinated adults and children who were diagnosed with COVID-19 from December 2019 through March 2021. Among those studied, 79% were hospitalized, and most patients (79%) lived in high-income countries. Patients’ median age was 54, and the majority of individuals (56%) were male.

The researchers analyzed patients’ health post-COVID during three intervals at one month (short-term), two to five months (intermediate-term) and six or more months (long-term).

According to the findings, survivors experienced an array of residual health issues associated with COVID-19. Generally, these complications affected a patient’s general well-being, their mobility or organ systems. Overall, one in two survivors experienced long-term COVID manifestations. The rates remained largely constant from one month through six or more months after their initial illness.

Did a black hole eating a star generate a neutrino?

Artist’s illustration of tidal disruption event AT2019dsg where a supermassive black hole spaghettifies and gobbles down a star. Some of the material is not consumed by the black hole and is flung back out into space.
Credit: DESY, Science Communication Lab

In October 2019, a high-energy neutrino slammed into Antarctica. The neutrino, which was remarkably hard to detect, piqued astronomers’ interest: what could generate such a powerful particle?

Researchers traced the neutrino back to a supermassive black hole that had just ripped apart and swallowed a star. Known as a tidal disruption event (TDE), AT2019dsg occurred just months earlier — in April 2019 — in the same region of the sky where the neutrino had come from. The monstrously violent event must have been the source of the powerful particle, astronomers said.

But new research casts doubt on that claim.

In a study published this month in the Astrophysical Journal, researchers at Northwestern University and the Center for Astrophysics | Harvard & Smithsonian, present extensive new radio observations and data on AT2019dsg, enabling the team to calculate the energy emitted by the event. The findings show AT2019dsg generated nowhere near the energy needed for the neutrino; in fact, what it spewed out was quite “ordinary,” the team concludes.