In the late 1970s, it was predicted that gene therapy would be applied to humans within a
decade. However, despite some success, gene therapy has still not become a routine …

Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …

Although a large percentage of the world population is seropositive for exposure to various
strains of adeno-associated virus (AAV), a human parvovirus, AAV has never been identified …

Introduction Gene therapy provides the exciting opportunity of a curative single treatment for
devastating diseases, eradicating the need for chronic medication. Adeno-associated …

Adenoviral, retroviral/lentiviral, adeno-associated viral, and herpesviral vectors are the major
viral vectors used in gene therapy. Compared with non-viral methods, viruses are highly …

Adeno-associated virus type 2 (AAV) is known to establish latency by preferential integration
in human chromosome 19q13. 42. The AAV non-structural protein Rep appears to target a …

In a recent editorial in Molecular Therapy, Büning and Schmidt1 discussed the role of gene
therapy vectors based on adeno-associated viruses (AAVs) in the development of liver …

Background: Ataxia telangiectasia (AT) is a rare autosomal-recessive multisystem disorder
characterized by pronounced cerebellar ataxia, telangiectasia, cancer predisposition and …

Achieving transgene integration into preselected genomic sites is currently one of the central
tasks in stem cell gene therapy. A strategy to mediate such targeted integration involves site …

Since its emergence onto the gene therapy scene nearly 25 years ago, the replication-
defective Herpes Simplex Virus Type-1 (HSV-1) amplicon has gained significance as a …