The Medicine Maker | mRNA Expands into Fertility Medicine

Credit: Mito Kanatsu-Shinohara Researchers in Japan have demonstrated that messenger RNA (mRNA) therapy can restore fertility in a genetic mouse model of male infertility – offering a potential new direction for reproductive medicine that avoids permanent gene editing. The study, published in Stem Cell Reports, shows that delivering therapeutic mRNA directly into … Read more

Gene-modified pancreas cells offer hope for people with type 1 diabetes

  Key Insights A proof-of-concept trial in a single patient has shown that genetically engineered pancreas cells can survive transplantation without immunosuppression. The results represent a watershed moment for both researchers and people with type 1 diabetes. Hurdles remain in taking the technology through to approved treatment. Four weeks after transplanting genetically modified… Source link

Genetic mismatch identified as major risk factor in cord blood transplantation

A Japanese study of more than 7,400 patients has identified a genetic mismatch that sharply increases the risk of severe acute graft-versus-host disease (GVHD) following umbilical cord blood transplantation. The specific donor–recipient human leukocyte antigen mismatch triples the likelihood of life-threatening immune complications, and severe acute GVHD itself is associated with significantly worse survival. These … Read more

First topical CRISPR gene therapy corrects disease-causing mutations in human skin

Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the University of British Columbia are extending those possibilities to the skin for the first time. The UBC team, together with researchers from the Berlin Institute of Health at Charité in Germany, has developed the first gene therapy capable of … Read more

Gene therapy targeting connexin 43 shows promise across inherited heart diseases

Jan 26 2026 University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse model can dramatically improve heart function and extend survival in several inherited forms of arrhythmogenic cardiomyopathy (ACM). The research suggests that a single gene therapy might someday help a wide range of ACM … Read more

Alzheimer’s gene therapy could help protect the brain from damage and preserve cognitive function

Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer’s disease that could help protect the brain from damage and preserve cognitive function. Unlike existing treatments for Alzheimer’s that target unhealthy protein deposits in the brain, the new approach could help address the root cause of Alzheimer’s disease … Read more

Gene Therapy For Inherited Disease In The Unborn Child

Until recently, even the most advanced gene therapies could only be given after a child was … More born—often racing against time to prevent irreversible damage. Image by freepik In the first part of this series, we explored how early genetic screening and gene therapy transform the lives of newborns and their families. Now, we’re … Read more

Baby’s Rare Metabolic Disease Cured with CRISPR

Archyde: Latest World News, Economy, Entertainment, Health, Technology & Sports Updates “`html Personalized Gene Therapy: A New Era for Rare Diseases Personalized Gene Therapy: A New Era for Rare Diseases Imagine a future where genetic diseases,once considered incurable,can be precisely targeted and treated with therapies tailored to an individual’s unique genetic makeup. This future is … Read more

Novartis Hires Marketing Assistant in the US

Novartis Seeks Marketing Assistant for Haematology Team in London CITY – May 3, 2024 – Global healthcare leader Novartis is hiring a Marketing Assistant for their haematology team in London, as announced today. Teh role, based in London, aims to support the company’s efforts in delivering value to customers and patients. The primary responsibilities will … Read more

First-in-Human Dosing: Key Preclinical Insights

“`html { “@context”: “https://schema.org”, “@type”: “NewsArticle”, “headline”: “Genespire Advances Gene Therapy for MMA: Preclinical Data unveiled”, “description”: “Genespire announced positive preclinical results for its gene therapy, marking a important step forward.”, “datePublished”: Genespire Advances Gene Therapy for MMA: Preclinical data Unveiled Gene Therapy Breakthrough for Methylmalonic Acidemia Milan, Italy – Genespire, a biotechnology firm specializing … Read more