SPAIN—For many Filipinos, diseases like heart failure and Parkinson’s disease remain life-changing diagnoses with few lasting solutions.Current ...

When gene therapy was first proposed in the 1970s, the idea seemed straightforward but revolutionary: replacing defective genes with functional ones to cure a disease. But over the decades, this ...

KaDee Troop is the mother of seven adopted children, four of whom have a rare genetic disorder that causes their skin to blister and tear at the slightest touch. Wounds heal slowly — if at all — and ...

In an update from uniQure, they report that their experimental gene therapy, AMT-130, has the potential to slow Huntington’s ...

Gene therapy appears to be a promising approach for a subset of genetic deafness, although challenges remain with development and manufacturing. Three major gene therapy approaches to address ...

This article is part of Nature Outlook: Skin, an editorially independent supplement produced with financial support from LEO Pharma. About this content. KaDee Troop is the mother of seven adopted ...

AMT-130 gene therapy slowed Huntington’s progression by 75%, offering the first evidence of a disease-modifying treatment.

Taysha Gene Therapies Inc (NASDAQ:TSHA) saw its stock leap 39% in premarket trading on Thursday after the company revealed that its gene therapy for Rett syndrome received Breakthrough Therapy ...

DNA vectors play an important role in gene therapy, serving as the vehicles that deliver genetic material into cells. While plasmid DNA (pDNA) is traditionally used, it has drawbacks that can limit ...

FOLLY BEACH — His pale blue eyes flashing and sporting a wide grin, Max Harbin toddled forward, leaning on a small cart as he ventured a few steps. His parents, Jim and Laura Harbin, watched him ...