Expression Targeting in Gene Therapy


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


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Further Reading

Clary BM and Lyerly HK (1998) Transcriptional targeting for cancer gene therapy. Surgical Oncology Clinics of North America 7: 565–574.

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.

Spear MA (1998) Gene therapy of gliomas: receptor and transcriptional targeting. Anticancer Research 18: 3223–3231.

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

Cystic fibrosis transmembrane conductance regulator, ATP‐binding cassette (sub‐family C, member 7) (CFTR); Locus ID: 1080. LocusLink:

Mucin 1, transmembrane (MUC1); Locus ID: 4582. LocusLink:

v‐erb‐b2 erythroblastic leukemia viral oncogene homolog 2, neuro/glioblastoma derived oncogene homolog (avian) (ERBB2); Locus ID: 2064. LocusLink:

Cystic fibrosis transmembrane conductance regulator, ATP‐binding cassette (sub‐family C, member 7) (CFTR); MIM number: 602421. OMIM:‐post/Omim/dispmim?602421

Mucin 1, transmembrane (MUC1); MIM number: 158340. OMIM:‐post/Omim/dispmim?158340

v‐erb‐b2 erythroblastic leukemia viral oncogene homolog 2, neuro/glioblastoma derived oncogene homolog (avian) (ERBB2); MIM number: 164870. OMIM:‐post/Omim/dispmim?164870

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How to Cite close
Vassaux, George, and Leverton, Kate E(Jan 2006) Expression Targeting in Gene Therapy. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1038/npg.els.0005747]