Homozygous familial hypercholesterolaemia affects approximately 30 000 individuals worldwide.1,2 Patients have untreated high ...

Viralgen and Axovia Therapeutics are partnering to develop and manufacture a gene therapy for the treatment of retinal ...

The companies will focus on advancing AXV-101, with which they hope to treat retinal dystrophy by targeting mutations in the BBS1 gene.

Axovia’s lead program AXV101 is an adeno-associated virus (AAV9)-based investigational gene therapy that aims to slow down or ...

Viralgen and Axovia Therapeutics are joining forces to advance the development and manufacture of an AAV9-based investigational gene therapy aimed at treating retinal dystrophy in patients with Bardet ...

1Department of Pathology, University of Texas Southwestern Medical Center, Dallas, Texas. 2Hamon Center for Therapeutic Oncology Research, University of Texas Southwestern Medical Center, Dallas, ...

The researchers found that all but one patient with cYNS had biallelic variants in CELSR1. The remaining patient had a heterozygous loss-of-function variant in FZD6 . Both CELSR1 and FZD6 are core ...

Collins and Talkowski provide a broad overview of structural variation in the human genome that covers their mutational properties, the dynamics of population genetics and functional consequences ...