More than two decades have passed since genetically modified HIV was used for gene
delivery. Through continuous improvements these early marker gene-carrying HIVs have …

The art and science of gene therapy has received much attention of late. The tragic death of
18-year-old Jesse Gelsinger, a volunteer in a Phase I clinical trial, has overshadowed the …

Mechanisms underlying seizure generation are traditionally thought to act over seconds to
minutes before clinical seizure onset. We analyzed continuous 3-to 14-day intracranial EEG …

▪ Abstract Broadly defined, the concept of gene therapy involves the transfer of genetic
material into a cell, tissue, or whole organ, with the goal of curing a disease or at least …

Neurotrophic factors are proteins with considerable potential in the treatment of central
nervous system (CNS) diseases and traumatic injuries. However, a significant challenge to …

The capacity to efficiently transduce nondividing cells, shuttle large genetic payloads, and
maintain stable long-term transgene expression are attributes that have brought lentiviral …

The central nervous system (CNS) is a sanctuary site and is protected by various barriers.
These regulate brain homeostasis and the transport of endogenous and exogenous …

Gene therapy can be broadly defined as the transfer of genetic material to cure a disease or
at least to improve the clinical status of a patient. One of the basic concepts of gene therapy …

RNAi is proving to be a powerful experimental tool for the functional annotation of
mammalian genomes. The full potential of this technology will be realized through …

Drug-inducible systems allowing the control of gene expression in mammalian cells are
invaluable tools for genetic research, and could also fulfill essential roles in gene-and cell …