Advances in vector-mediated gene transfer.
Clinical applications of gene transfer technology initially targeted the treatment of inherited monogenetic disorders and cancers refractory to conventional therapies. Today, gene transfer approaches are being developed for most tissues and for multiple disorders including those affecting quality of life. The focus herein is eventual application of gene transfer technology for the management of organ-directed autoimmunity. A specific example is presented: Sjögren's syndrome and localized salivary gland gene transfer. The status of relevant pre-clinical gene transfer studies is reviewed, with an emphasis on use of adenoviral and adeno-associated viral vectors. Current limitations of effective organ-directed gene transfer are also discussed.
Baum
Bruce J
BJ
Gene Therapy and Therapeutics Branch, National Institute of Dental and Craniofacial Research, NIH, DHHS, Building 10, Room 1N113, MSC-1190, Bethesda, MD 20892-1190, USA. bbaum@dir.nidcr.nih.gov
Goldsmith
Corinne M
CM
Kok
Marc R
MR
Lodde
Beatrijs M
BM
van Mello
Norah M
NM
Voutetakis
Antonis
A
Wang
Jianghua
J
Yamano
Seiichi
S
Zheng
Changyu
C
D000256:Adenoviridae / Q000235:genetics; D002986:Clinical Trials as Topic; D018014:Gene Transfer Techniques / Q000639:trends; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics*; D006801:Humans; D012859:Sjogren's Syndrome / Q000235:genetics / Q000628:therapy