Queen’s researchers working on gene therapy answer to rare, fatal genetic disorder

Dr. Jagdeep Walia is a medical geneticist and full-time professor in the Division of Medical...

Intracerebroventricular SPAST-AAV9 gene therapy prevents manifestation of symptoms in a mouse model of SPG4 hereditary spastic paraplegia

Hereditary spastic paraplegia type 4 is characterized by gait impairments, progressive spasticity, and weakness of...

Person previously ineligible for gene therapy granted treatment after antibody-chopping drug clears the way

Ryan Cross Senior Science Correspondent SEVILLE, SPAIN — Gene therapies are offering new hope to...

Patient groups push abandoned rare disease gene therapy toward clinical trial

Two rare disease groups have produced the key funding needed to make a gene therapy...

Published in Nature Medicine: Breakthrough gene therapy trial for Tay-Sachs Disease

Blu Genes Foundation supported: Duel-vector rAAVrh8 gene therapy for GM2 gangliosidosis: a phase I/II trial...

Baby Is Healed With World’s First Personalized Gene-Editing Treatment

Article: By Gina Kolata, The New York Times – May 15, 2025 The technique used...

Study identifies a potential treatment for Sandhoff and Tay-Sachs diseases

In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff...

Blu Genes supports gene therapy at University of Wisconsin-Madison. Gene therapy protects against motor neuron disease.

Anjon Audhya The mutation is in the Trk-fused gene, which typically facilitates the transportation of...

Taylor Swift tickets being auctioned off to help fund for Ottawa boy with rare disease

A pair of Taylor Swift tickets are being auctioned off in Ottawa to help fund...

Saskatchewan artist fundraising to help family whose son has genetic disease

A Regina based artist is using his platform to fundraise in hope of helping a...