All posts tagged: Gene Therapy

Salamander gene could hold the key to regrowing human limbs

Salamander gene could hold the key to regrowing human limbs

Published by Jenni Sidey

The skin over a fresh wound might not look like much. In some animals, though, that thin covering becomes command central for rebuilding what was lost. That idea sits at the heart of new research on axolotls, zebrafish, and mice, three species that are very different on the surface but share part of the same genetic machinery when they regenerate damaged body parts. By tracing that overlap, scientists say they have found an early clue toward a gene therapy strategy that might someday help people regrow complex tissues after injury. “This significant research brought together three labs, working across three organisms to compare regeneration,” said Josh Currie, an assistant professor of biology at Wake Forest whose lab studies the Mexican axolotl salamander. “It showed us that there are universal, unifying genetic programs that are driving regeneration in very different types of organisms, salamanders, zebrafish and mice.” The work brought together Currie, Duke University plastic surgeon David A. Brown, and Kenneth D. Poss of the University of Wisconsin-Madison. Their shared target was limb and appendage regeneration, …

New genetic test predicts lung cancer spread before surgery

New genetic test predicts lung cancer spread before surgery

Published by Jenni Sidey

A quiet signal inside a tumor may soon guide how surgeons treat early lung cancer. Researchers now report that hidden genetic patterns can reveal whether a tumor has already begun to spread through blood vessels, even before surgery begins. That discovery could change how doctors approach lung adenocarcinoma, the most common form of lung cancer in the United States. It may also help answer a painful question that many patients face after surgery: will the cancer come back? A Hidden Threat Inside Early Tumors Lung cancer remains the leading cause of cancer-related death. It kills more people in the United States than breast, prostate, and colon cancers combined. Even when doctors detect it early, outcomes can vary widely. Study overview and distinct gene expression changes associated with VI in stage I LUAD. (CREDIT: Nature Communications) Some tumors behave quietly and never return after removal. Others come back months or years later, often more aggressive than before. One of the strongest warning signs is vascular invasion. Vascular invasion occurs when cancer cells break into nearby blood …

Researchers create new molecule to insert DNA into cells

Researchers create new molecule to insert DNA into cells

Published by Jenni Sidey

Getting DNA into a living cell sounds simple, until you remember the cell’s outer membrane acts like a guarded wall. DNA strands carry a negative charge, and they do not cross that wall easily. So for years, many genetic therapies and DNA-based vaccines have depended on delivery helpers that are strongly positively charged, because opposite charges stick. That trick works, but it can come with baggage: irritation, inflammation, and messy clumping with other molecules in the body. A team led by Professor Shoichiro Asayama at Tokyo Metropolitan University set out to dodge that tradeoff. Their approach uses an uncharged polymer, paired with a small DNA “handle,” to escort plasmid DNA into cells. In mouse experiments, their best formulation produced luciferase gene expression that tended to be about 14 times higher than annealed DNA delivered without a carrier, with results reported as p < 0.1 in groups of four mice. The goal is not a new gene-editing tool. It is a new way to carry DNA safely, particularly into skeletal muscle, where the surrounding environment can …

Single protein is key to treating a deadly genetic heart disease targeting young athletes

Single protein is key to treating a deadly genetic heart disease targeting young athletes

Published by Jenni Sidey

A research team at the University of California San Diego has discovered a novel and promising method of treating arrhythmogenic cardiomyopathy (ACM), a rare inherited heart disease that can strike suddenly and violently, developing into a life-threatening condition in young, healthy athletes. By restoring expression of an important protein, connexin-43, in murine (mouse) models, the investigators dramatically improved overall heart function, significantly reduced dangerous heart arrhythmias, and increased survival rates by greater than twofold. Patients with ACM do not have the ability to efficiently pump blood due to damage to their cell-to-cell contact proteins, known as desmosomes, which are weakened by genetic defects. Furthermore, patients with ACM experience an increased risk for sudden cardiac death, particularly athletes, because of the additional stress placed on an already damaged, structurally weak heart by prolonged, high levels of activity. Results of this study, published in the journal Circulation: Heart Failure, indicate that one potential gene-based therapy will offer hope for the treatment of many different types of patients with this condition. This benefit appears regardless of the underlying …