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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 partnering to develop and manufacture a gene therapy for the treatment of retinal ...

R package providing a tidy grammar of population genetics, facilitating the manipulation and analysis of data on biallelic single nucleotide polymorphisms (SNPs). R package providing a tidy grammar of ...

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 ...

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

Clinical genetics involves the study, counselling and treatment of individuals and families with heritable disorders and disease predisposition. Diagnostic tools include standard ontologies for ...

Humans are the only mammals with long hair on their heads. Scientists look into what drives this unique feature.

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

By contrast, among relatives who were heterozygous for a mutation in JAK1 (c.2099G>A; p.S700N), the unaffected carrier T cells predominantly expressed the wild-type JAK1 allele, whereas the affected ...

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