▪ Abstract The driving interest in adeno-associated virus (AAV) has been its potential as a
gene delivery vector. The early observation that AAV can establish a latent infection by …

Our ability to manipulate the genetic constitution of the nervous system has come of age with
various technologies, including virus vectors that can efficiently deliver genes to neurons …

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 …

TorsinA is an AAA+ protein located predominantly in the lumen of the endoplasmic reticulum
(ER) and nuclear envelope responsible for early onset torsion dystonia (DYT1). Most cases …

Our goal is the development of in vivo hematopoietic stem cell (HSC) transduction
technology with targeted integration. To achieve this, we modified helper-dependent …

Adeno-associated viruses (AAV) are widely spread throughout the human population, yet no
pathology has been associated with infection. This fact, together with the availability of …

There are two types of herpes simplex virus type 1 (HSV-1) vector systems in gene therapy,
termed amplicon and recombinant virus [1–5]. HSV-1 amplicon vectors represent the first …

Amplicons are defective and non-integrative vectors derived from herpes simplex virus type
1. As the vector genome carries no virus genes, amplicons are both non-toxic for the infected …

The modification of mammalian genomes is an important goal in gene therapy and animal
transgenesis. To generate stable genetic and biochemical changes, the therapeutic genes …

HSV-1 amplicons can accommodate foreign DNA of any size up to 150 kbp. Genomic
sequences as well as cDNA, large transcriptional regulatory sequences for cell type-specific …