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.