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Category: biotech/medical – Page 15

Three-parent babies: Mitochondrial replacement therapies

The mitochondria are membrane-bound intracellular organelles present in almost all eukaryotic cells (). They generate energy through oxidative phosphorylation, and are responsible for 90% of cellular ATP (). In mammals, the mitochondria are present in all cells, except the enucleated red blood cells, being more present in tissues that need energy metabolism, with several units of the organelle. They have a round or oval shape and are about 0.5 to 1 µm in diameter, and up to 7 µm in length (). Together with the cell nucleus, they are the only cell organelle having their own genome, an extremely compact molecule, with 16.500 base pairs and 37 genes: 13 messenger RNAs, 22 RNAs, and 2 ribosomal RNAs. The D-loop is the only non-coding region in mtDNA, since introns and intergenic regions are non-existent or restricted to a few nucleotides ().

In addition to the production of reactive oxygen species due to the release of free electrons generated from the respiratory chain, mitochondria have few repair systems and therefore are subject to genetic mutations, causing diseases that affect approximately 1 in 5,000 people (). Mitochondrial diseases can affect organs that depend on energy metabolism, such as skeletal muscle, cardiac, central nervous system, endocrine, retina and liver (; ), giving rise to several incurable diseases, such as: deafness, diabetes mellitus, myopathies, glaucoma and others (). These metabolic disorders, lead to inefficient oxidative phosphorylation, impairing cell energy production (). They are difficult to diagnose and most of the time untreated, affecting adults and children ().

Mitochondria are inherited only from the female gamete; therefore, the mitochondrial DNA is of exclusive maternal inheritance (). The genetic mutations present in this material can be avoided using mitochondrial substitution techniques (), where the nuclear genome is withdrawn from an oocyte, which carries mitochondrial mutations, and is implanted in a normal enucleated donor ().

Altered microbiome: Oral bacteria play a role in chronic liver disease, study reveals

Each year, more than two million people die from advanced chronic liver disease (ACLD). Previous research has linked gut microbiome disruptions to this condition and suggested that bacteria typically found in the mouth may colonize the gut.

A new study published in Nature Microbiology now shows that identical bacterial strains occur in both the mouth and gut of patients with advanced chronic liver disease and also reveals a mechanism by which oral bacteria affect gut health. The researchers also found that this process coincides with worsening liver health.

Simulations predict antibody drug viscosity, paving way for easier self-injection

Monoclonal antibodies (mAbs) aid the body against autoimmune diseases and cancer, among other things. Patients have to pick up the medicine every few weeks. It would be easier for them to be able to inject the medicine themselves at home, but this would only be possible if the medications were highly concentrated but not too viscous.

A team at Ruhr University Bochum, Germany, led by Professor Lars Schäfer from the Center for Theoretical Chemistry and the company Boehringer Ingelheim Pharma have developed a quick and realistic simulation method to make this possible. This method can predict how formulations will behave.

The team reports its findings in the Journal of Physical Chemistry.

First breathing ‘lung-on-chip’ developed using genetically identical cells

Researchers at the Francis Crick Institute and AlveoliX have developed the first human lung-on-chip model using stem cells taken from only one person. These chips simulate breathing motions and lung disease in an individual, holding promise for testing treatments for infections like tuberculosis (TB) and delivering personalized medicine.

The research is published in the journal Science Advances.

Air sacs in the lungs called alveoli are the essential site of gas exchange and also an important barrier against inhaled viruses and bacteria that cause respiratory diseases like flu or TB.

Penn engineers turn toxic fungus into anti-cancer compound

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Penn-led researchers have turned a deadly fungus into a potent cancer-fighting compound. After isolating a new class of molecules from Aspergillus flavus, a toxic crop fungus linked to deaths in the excavations of ancient tombs, the researchers modified the chemicals and tested them against leukemia cells. The result? A promising cancer-killing compound that rivals FDA-approved drugs and opens up new frontiers in the discovery of more fungal medicines.

“Fungi gave us penicillin,” says Sherry Gao, Presidential Penn Compact Associate Professor in Chemical and Biomolecular Engineering (CBE) and in Bioengineering (BE) and senior author of a new paper in Nature Chemical Biology on the findings. “These results show that many more medicines derived from natural products remain to be found.”

How Fat Type Shapes Hypertension Risk

Too much fat can raise blood pressure, but the type matters more than the amount.

Researchers at Rockefeller University uncovered how thermogenic fat keeps blood vessels flexible by shutting down a vessel-stiffening enzyme.

Read more.

Promoting brown fat activity could counteract hypertension by reshaping the molecular signals that govern vascular stiffness.

Scientists turn cells’ most mysterious structures into spies on genetic activity

The barrel-shaped structures found by the thousands in most animal cells are one of biology’s biggest mysteries. But although researchers haven’t figured out the function of these “vaults,” they now report a new use for the puzzling particles.

Enigmatic ‘vaults’ can be engineered to eavesdrop on RNA, aiding cancer studies and more.

A breakthrough in DNA sequencing hints at why most smokers don’t get lung cancer

Breakthrough in DNA sequencing offers clues to why most smokers do not develop lung cancer.

“Our data suggest that these individuals may have survived for so long in spite of their heavy smoking because they managed to suppress further mutation accumulation,” says pulmonologist and genetics researcher Simon Spivack, a co-author on the study. “This leveling off of mutations could stem from these people having very proficient systems for repairing DNA damage or detoxifying cigarette smoke.”

Researchers who study the health effects of cigarette smoke have used all kinds of methods — from giving lab animals high doses of chemicals found in tobacco to combing through archives to determine which diseases smokers get more often — to figure out how the habit affects the body. Those studies have made it clear that cigarettes contain hundreds of harmful chemicals, including dozens of carcinogens.

For decades, researchers didn’t have any way to measure the mutations in lung cells that actually cause lung cancer. Five years ago, researchers at Albert Einstein College of Medicine in New York found a way to overcome technical limitations that had made it impossible to sequence the genome. That is, they figured out how to determine the exact order of the A, T, C, and G molecules of the DNA within a single cell without introducing too many errors in the process.

Frontline Osimertinib Combo Prolongs Survival in Advanced EGFR+ NSCLC

Among patients with central nervous system (CNS) metastases at baseline, the 36-month OS rate was 57% (95% CI, 48%-66%) with osimertinib/chemotherapy and 40% (95% CI, 31%-49%) with osimertinib alone. The 36-month OS rates among patients without CNS metastases were 67% (95% CI, 59%-74%) vs 58% (95% CI, 50%-65%) in each respective arm. Additionally, the 36-month rates were 54% (95% CI, 44%-63%) vs 42% (95% CI, 32%-51%) among patients with EGFR L858R mutations and 69% (95% CI, 61%-75%) vs 57% (95% CI, 49%-64%) among those with EGFR exon 19 deletions.

When considering patients who discontinued frontline osimertinib following disease progression, 69% (n = 88/127) of patients in the combination therapy arm and 77% (n = 143/185) in the monotherapy arm received subsequent therapy. The most common types of first subsequent therapy in the combination arm included platinum-based chemotherapy (44%) and non–platinum-based chemotherapy (30%). Among patients who received osimertinib monotherapy, the most common kind of subsequent treatment was platinum-containing chemotherapy (72%).

“The combination therapy used in this trial was associated with a higher incidence of grade 3 or higher adverse events and of [AEs] leading to the discontinuation of treatment than osimertinib monotherapy. Most high-grade toxic effects associated with the combination therapy were related to myelosuppressive effects, which are generally dose-related and reversible, with supportive interventions available to ameliorate such effects,” lead study author Pasi A. Jänne, MD, PhD, senior vice president of Translational Medicine and professor in the Department of Medical Oncology at Lowe Center for Thoracic Oncology of Dana–Farber Cancer Institute, wrote with coauthors in the publication.1 “Results from this trial provide evidence that first-line treatment with osimertinib plus platinum/pemetrexed led to significantly longer [OS] than osimertinib monotherapy among patients with EGFR-mutated advanced NSCLC.”

Metformin hijacks AMPK-ERK1/2 signaling to trigger a pathogenic “selection trap” and thymic atrophy

Qin et al. reveal that the diabetes drug metformin sets a “selection trap” in the thymus. It pushes developing immune cells toward maturation while simultaneously triggering their apoptosis via a metabolic stress-induced pathway, challenging the drug’s safety in non-diabetic contexts.

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