title: Gene therapy: therapeutic applications and relevance to pathology.
abstract: This review discusses gene therapy as a new treatment paradigm where genetic material is introduced into cells for therapeutic benefit. The genetic material is the 'drug'. It can have a transient or ongoing effect depending on whether or not the introduced genetic material becomes part of the host cell DNA. Different delivery and gene technologies are chosen by investigators to maximise gene delivery to, and expression within, the target cells appropriate for the disease indication. The presence and expression of the introduced genetic material is monitored by molecular means so that treatment efficacy can be assessed via changes in surrogate and/or actual markers of disease. Of interest to the pathologist will be the approaches being developed for the disease indications highlighted and the monitoring of treatment efficacy.
authors: Broadvector Limited, North Ryde, Western Australia, Australia.
authors: Gerald
authors: G
authors: Both
authors: Ian
authors: I
authors: Alexander
authors: Sue
authors: S
authors: Fletcher
authors: Tamara J
authors: TJ
authors: Nicolson
authors: John E J
authors: JE
authors: Rasko
authors: Steve D
authors: SD
authors: Wilton
authors: Geoff
authors: G
authors: Symonds
mesh_terms: D004353:Drug Evaluation, Preclinical; D015316:Genetic Therapy; D005822:Genetic Vectors; D015658:HIV Infections / Q000235:genetics / Q000628:therapy; D019337:Hematologic Neoplasms / Q000235:genetics / Q000628:therapy; D006801:Humans; D008297:Male; D010336:Pathology / Q000379:methods*; D011471:Prostatic Neoplasms / Q000235:genetics / Q000628:therapy