Expression Targeting in Gene Therapy

George Vassaux, Imperial College at Hammersmith Hospital, London, UK
Kate E Leverton, Imperial College at Hammersmith Hospital, London, UK

Published online: January 2006

DOI: 10.1038/npg.els.0005747

Expression targeting involves the use of tissue-specific enhancer/promoter elements to target the expression of therapeutic transgenes to tissues of interest. With the development of in vivo delivery vectors, these selective expression cassettes can now be used for preclinical assessment of their therapeutic potential for the treatment of inherited diseases or cancers.

Keywords: transcription; targeting; selectivity; gene therapy; expression cassette

 References
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    Larochelle N, Lochmuller H, Zhao J, et al. (1997) Efficient muscle-specific transgene expression after adenovirus-mediated gene transfer in mice using a 1.35 kb muscle creatine kinase promoter/enhancer. Gene Therapy 4: 465–472.
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    Clary BM and Lyerly HK (1998) Transcriptional targeting for cancer gene therapy. Surgical Oncology Clinics of North America 7: 565–574.
    book Lemoine NR (1999) Understanding Gene Therapy. Oxford: BIOS Scientific.
    Segal DJ and Barbas 3rd CF (2000) Design of novel sequence-specific DNA-binding protein. Current Opinion in Chemical Biology 4: 34–39.
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    Stone D, David A, Bolognani F, Lowenstein PR and Castro MG (2000) Viral vectors for gene delivery and gene therapy within the endocrine system. Journal of Endocrinology 164: 103–118.
 Web Links
    ePath Cystic fibrosis transmembrane conductance regulator, ATP-binding cassette (sub-family C, member 7) (CFTR); Locus ID: 1080. LocusLink: http://www.ncbi.nlm.nih.gov/LocusLink/LocRpt.cgi?l=1080
    ePath Mucin 1, transmembrane (MUC1); Locus ID: 4582. LocusLink: http://www.ncbi.nlm.nih.gov/LocusLink/LocRpt.cgi?l=4582
    ePath v-erb-b2 erythroblastic leukemia viral oncogene homolog 2, neuro/glioblastoma derived oncogene homolog (avian) (ERBB2); Locus ID: 2064. LocusLink: http://www.ncbi.nlm.nih.gov/LocusLink/LocRpt.cgi?l=2064
    ePath Cystic fibrosis transmembrane conductance regulator, ATP-binding cassette (sub-family C, member 7) (CFTR); MIM number: 602421. OMIM: http://www.ncbi.nlm.nih.gov/htbin-post/Omim/dispmim?602421
    ePath Mucin 1, transmembrane (MUC1); MIM number: 158340. OMIM: http://www.ncbi.nlm.nih.gov/htbin-post/Omim/dispmim?158340
    ePath v-erb-b2 erythroblastic leukemia viral oncogene homolog 2, neuro/glioblastoma derived oncogene homolog (avian) (ERBB2); MIM number: 164870. OMIM: http://www.ncbi.nlm.nih.gov/htbin-post/Omim/dispmim?164870

Related Sub-Topics:

Techniques and Tools in Clinical Genetics | Gene Therapy | Transcriptional Regulation
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Article Title: Expression Targeting in Gene Therapy
 
How to Cite close
Vassaux, George, and Leverton, Kate E(Jan 2006) Expression Targeting in Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. http://www.els.net [doi: 10.1038/npg.els.0005747]