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Category: genetics – Page 199

First-of-Its-Kind Gene Therapy Can Be Applied to Skin Instead of Injected

The root cause is frustratingly simple: one gene mutation, which affects a critical protein that helps support skin integrity. The single genetic error makes the illness a perfect candidate for gene therapy. Yet with the skin already fragile, injections—a current standard for gene therapy—are hard to tolerate.

What about a genetic moisturizer instead?

This month, the FDA approved the first rub-on gene therapy. Similar to aloe vera for treating sunburns, the therapy comes in a gel that’s gently massaged onto blisters and wounds to help with healing. Dubbed Vyjuvek, it directly delivers healthy copies of the mutated gene onto damaged skin. An alternative version is configured into eye drops to reconstruct the eye’s delicate architecture to better support sight.

Unveiling a Genetic Breakthrough: Enhancing Cancer-Killing Cells and Extending Lifespan

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In the pursuit of extending healthy human lifespans, scientists have achieved a remarkable breakthrough that marks a significant milestone in the field. Researchers from Taipei Medical University in Taiwan have uncovered a genetic modification in mice that can empower cancer-killing cells, increasing their effectiveness by two to seven times while extending their lifespan by up to 20 percent.

Building upon last year’s groundbreaking study, the scientists have now successfully replicated these extraordinary outcomes in ordinary mice through a single transplant of blood stem cells. The findings, published in the scientific journal Cold Spring Harbor Protocols, hold immense importance, according to Che-Kun James Shen, the lead researcher of the study. He believes that these findings could have profound implications for human health and anticipates that clinical trials could commence as early as the end of this year or next year.

The initial discovery involved identifying an amino acid, known as KLF1, that, when altered, preserves the youthful characteristics of the mice. This includes improved motor function, enhanced learning and memory, as well as more effective anti-cancer cells. Additionally, the mice exhibited darker and shinier hair, and a significant reduction in fibrosis—a process associated with impaired organ functioning that occurs during aging.

The Woman Who Doesn’t Feel Pain — New Study Reveals Her Unique Molecular Machinery

New research from UCL, investigating the biology of a rare genetic mutation that enables carrier Jo Cameron to live virtually without pain and fear while also healing quickly, discovered that the mutation in FAAH-OUT gene ‘turns down’ FAAH gene expression, affecting molecular pathways related to wound healing and mood, thereby offering potential new targets for drug discovery.

New research from University College London (UCL) has unraveled the biology behind a unique genetic mutation that results in its carrier experiencing minimal pain, enhanced healing, and lower levels of anxiety and fear.

Published in the journal Brain, the research is a follow-up to the team’s 2019 discovery of the FAAH-OUT gene and its rare mutations, which make Jo Cameron almost immune to pain, and devoid of fear and anxiety. The latest study elucidates how this mutation reduces the expression of the FAAH gene and impacts other molecular pathways associated with mood and wound healing. The insights garnered from these findings could potentially pave the way for novel drug targets and foster further research in these domains.

Hope for age-related hearing loss with novel gene therapy

Age-related hearing loss impacts one in three adults between the ages of 64 and 75 in the US, and around half of these numbers are down to genes.

The extra kicker, though, is that because hearing involves a complex genetic toolkit, it also makes this kind of hearing loss incredibly difficult to treat.

A team of researchers has for the first time targeted age-related genetic hearing loss in a much older cohort of mice, which had a mutation of the human transmembrane serine protease 3 (TMPRSS3) gene that results in autosomal recessive deafness 8/10 (DFNB8/DFNB10).

The DNA of Decision-Making

Summary: A novel study uncovers a peculiar pattern of decision-making in mice, influenced by a specific gene named Arc.

While searching for food, mice repeatedly visited an empty location instead of staying at a site abundant in food. However, mice lacking the Arc gene demonstrated a more practical approach, sticking with the food-rich site, thereby consuming more calories overall.

This unique research potentially opens the door for a new field, ‘decision genetics’, investigating the genetic influence on decision-making, possibly even in humans.

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Gene Editing Gets a Triple Boost: “Happy Accident” Leads to Enhanced CRISPR Efficiency

Scientists have enhanced the efficiency of CRISPR/Cas9 gene editing by threefold using interstrand crosslinks, without resorting to viral material for delivery. This approach boosts the cell’s natural repair mechanisms, allowing for more accurate and efficient gene editing, potentially improving disease research and preclinical work.

Gene editing is a powerful method for both research and therapy. Since the advent of the Nobel Prize-winning CRISPR/Cas9 technology, a quick and accurate tool for genome editing discovered in 2012, scientists have been working to explore its capabilities and boost its performance.

Researchers in the University of California, Santa Barbara biologist Chris Richardson’s lab have added to that growing toolbox, with a method that increases the efficiency of CRISPR/Cas9 editing without the use of viral material to deliver the genetic template used to edit the target genetic sequence. According to their new paper published in the journal Nature Biotechnology, their method stimulates homology-directed repair (a step in the gene editing process) by approximately threefold “without increasing mutation frequencies or altering end-joining repair outcomes.”

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