What are Genes and what do they do?
Genes are segments of DNA (deoxyribonucleic acid) that carry the instructions your body uses to build and operate itself. They are the fundamental units of heredity, passed from parents to offspring.
Welcome to GenTher
GenTher is your go-to destination for the latest news, research, and insights on gene therapy. Our goal is to share important information and advancements in the field to help educate and inform our readers.
Educational Articles
Approved and Successful Gene Therapies
1. Spinal Muscular Atrophy (SMA)
Cystic Fibrosis Gene Therapy
Cystic fibrosis (CF) gene therapy is a medical approach that aims to treat the underlying genetic cause of cystic fibrosis rather than just managing its symptoms. CF is caused by mutations in the CFTR gene (Cystic Fibrosis Transmembrane Conductance Regulator), which leads to the production of a faulty CFTR protein responsible for regulating salt and water movement in and out of cells. This defect results in thick, sticky mucus buildup in the lungs and other organs.
What is Gene Therapy?
Gene therapy is a medical technique that involves altering a person’s genes to treat or prevent disease. It aims to correct defective genes responsible for disease development or to introduce new genes into the body to help fight a condition.
Research & Scientific Articles
New Gene Therapy Shows Promise in Fighting Solid Tumors - Scientists use a virus protein to attack cancer cells while leaving healthy cells unharmed
A new type of cancer treatment may be on the horizon—and it comes from an unlikely source: a virus. Researchers have found that a tiny part of a virus, called 3C protease, could be a powerful weapon against many types of solid tumors, which are cancers that form lumps in organs like the lungs, liver, or breast.
Correction of the most common Cystic Fibrosis mutation (F508del) using prime editing
Cystic fibrosis is primarily caused by the F508del mutation in the CFTR gene, leading to defective chloride ion transport in epithelial cells. Traditional treatments require lifelong administration of small-molecule drugs, which, while beneficial, do not offer a permanent solution. Prime editing (PE) emerges as a promising gene-editing tool capable of precise DNA modifications without inducing double-stranded breaks.
Ethics, Policy, and Regulation
Ethical Considerations in Gene Therapy
Gene therapy, the technique of altering genes within an individual’s cells to treat or prevent disease, holds transformative potential for medicine. From curing genetic disorders to enhancing immune responses to cancer, gene therapy presents unprecedented possibilities. However, its promise is accompanied by a complex web of ethical concerns that demand careful examination. As science races ahead, ethical deliberation must keep pace to ensure that gene therapy benefits society while minimizing harm and injustice.
News and updates
A Baby, a Mutation, and a Medical First: How Scientists Edited Genes Inside the Body to Save a Life
In a medical milestone that sounds like science fiction, doctors have successfully edited a baby's genes inside the body to treat a rare and deadly genetic disease—marking the first time such a personalized approach has been used in a human being.
A Smart Switch for Gene Therapy: How Scientists Are Fine-Tuning Treatments to Be Just Right
The scientific paper titled "Model-guided design of microRNA-based gene circuits supports precise dosage of transgenic cargoes into diverse primary cells" introduces an innovative approach to gene therapy, aiming to ensure that therapeutic genes are expressed at optimal levels within cells.
Biorxiv gene therapy articles (not peer-reviewed)
Gene Therapy Breakthrough Offers Hope for Children with Rare Brain Disorder Non-Ketotic Hyperglycinemia (Not peer-reviewed)
A team of scientists at the University of Notre Dame has developed a promising gene therapy that could offer life-saving treatment for children with a devastating rare condition known as Non-Ketotic Hyperglycinemia (NKH).
About us
At GenTher, we are passionate about exploring the potential of gene therapy and sharing valuable knowledge with our audience. Our team of experts is dedicated to providing accurate and up-to-date information on this cutting-edge field.