Concepts in genetic medicine

TL;DR: It is shown that RNAi can be applied to inhibit production of HBV replicative intermediates in cell culture and in immunocompetent and immunodeficient mice transfected with an HBV plasmid, showing that such an approach could be useful in the treatment of viral diseases.

TL;DR: In this paper, the authors reviewed T-cell products screened for replication-competent lentivirus (RCL) using methodology developed in the National Gene Vector Biorepository.

TL;DR: Advances in the technological approaches and methods for ex vivo manipulation have led to T lymphocytes endowed with enhanced potency and unique functions, with promise as the new generation of infused therapeutics.

TL;DR: In this article, the authors applied s-galactosidase expressing equine infectious anaemia virus (EIAV) pseudotyped with VSV-G by single or combined injection via different routes to the MF1 mouse fetus on day 15 of gestation and describe substantial gene delivery to the musculature.

TL;DR: It is shown, using luciferase and beta-galactosidase containing plasmids, that dendrimers mediate high efficiency transfection of a variety of suspension and adherent cultured mammalian cells.

TL;DR: It is suggested that NSC migration can be extensive, even in the adult brain and along nonstereotypical routes, if pathology is present, and the adjunctive use of inherently migratory NSCs as a delivery vehicle for targeting therapeutic genes and vectors to refractory, migratory, invasive brain tumors is suggested.

TL;DR: Tests for recombination events that might result in intact retrovirus showed no evidence for the generation of replication-competent virus and results suggest that gag, pol, and env, when present on different plasmids, may provide an efficient and safe packaging line for use in retroviral gene transfer.

TL;DR: It is found that an AAV vector containing the LacZ gene resulted in expression of β-galactosidase up to three months post-injection in vivo, and safe and stable TH gene transfer into the denervated striatum may have potential for the genetic therapy of Parkinson's disease.