Adeno-associated virus-mediated gene transfer

Kinetics of recombinant adeno-associated virus-mediated gene transfer.

Recombinant adeno-associated virus (rAAV) vectors have been shown to be useful for efficient gene delivery to a variety of dividing and nondividing cells. Mechanisms responsible for the long-term, persistent expression of the rAAV transgene are not well understood. In this study we investigated the kinetics of rAAV-mediated human factor IX (hFIX) gene transfer into human primary myoblasts and m...

Intrathecal gene transfer by adeno-associated virus for pain.

Chronic pain is among the most prevalent medical problems, affecting more than half of patients with advanced cancer and many with other common diseases. Current analgesics often fail to provide satisfactory symptom relief and frequently cause severe side effects. Intrathecal (IT) gene transfer is an attractive method for pain research in rodent models, because it allows targeting of a wide var...

Adeno-associated virus vectors for therapeutic gene transfer.

Adeno-associated virus (AAV) is currently being used in several human gene therapy trials, including one targeting hemophilia B and another targeting cystic fibrosis, and to date has demonstrated persistent expression without inflammation of the target tissue. Some of the unique features that have distinguished AAV from other gene therapy vectors include (i) its ability to transduce both dividi...

Supraspinal gene transfer by intrathecal adeno-associated virus serotype 5

We report the pattern of transgene expression across brain regions after intrathecal delivery of adeno-associated virus serotype 5 (AAV5). Labeling in hindbrain appeared to be primarily neuronal, and was detected in sensory nuclei of medulla, pontine nuclei, and all layers of cerebellar cortex. Expression in midbrain was minimal, and generally limited to isolated neurons and astrocytes in the c...

Transgene Expression Transfer: Role of Cellular FKBP52 Protein in Adeno-Associated Virus Type 2-Mediated Gene