Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

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 …

The ability of recombinant adeno-associated viral (rAAV) vectors to exhibit minimal
immunogenicity and little to no toxicity or inflammation while eliciting robust, multiyear gene …

Gene therapy involves the transfer of genetic information to a target cell to facilitate the
production of therapeutic proteins and is now a realistic prospect as a cancer treatment …

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 …

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 …

Ataxia-telangiectasia (AT) is an autosomal recessive disorder characterized by
neurodegeneration, immunodeficiency, cancer predisposition, genome instability, and …

Amplicons are defective and non-integrative vectors derived from herpes simplex virus type
1. They carry no virus genes in the vector genome and are, therefore, not toxic to the infected …

The last two years have seen significant advances in our understanding of the cellular
innate responses elicited or activated by the entry of amplicon particles, which may, in part …

Random integration of viral gene therapy vectors and subsequent activation or disruption of
cellular genes poses safety risks. Major efforts in the field are aimed toward targeting vector …