Strategies for achieving multiple layers of selectivity in gene therapy

Molecular Medicine Today
R G VileR M Diaz

Abstract

Here we review the progress towards the development of targeted vectors for direct in vivo delivery in gene therapy. Currently, there are many separate approaches. These include: simple physical/anatomical localization of administration of the vector at the site where gene transfer is required; exploitation of natural tropisms of plasmid, viral and cellular vectors; and the use of molecular engineering to change the specificity of proteins and nucleic acids so that they specifically recognize target ligands expressed on/in the target cells. Unfortunately, each of these approaches is usually imperfect by itself. However, combinations of these strategies might produce vectors in which several layers of imperfect targeting give an overall level of specificity that can justify systemic delivery of vectors to treat human disease.

References

Oct 1, 1992·Human Gene Therapy·F L Moolten, L A Cupples
Jan 1, 1994·European Journal of Cancer : Official Journal for European Organization for Research and Treatment of Cancer (EORTC) [and] European Association for Cancer Research (EACR)·S J Russell
Aug 25, 1995·Cell·R A Weiss, C S Tailor
Oct 1, 1993·Annals of Surgery·S A RosenbergS E Ettinghausen
Dec 1, 1993·Proceedings of the National Academy of Sciences of the United States of America·G J NabelA E Chang
Mar 1, 1993·AIDS Research and Human Retroviruses·M L GougeonL Montagnier
Apr 1, 1993·Trends in Genetics : TIG·N Dillon, F Grosveld
Jun 11, 1996·Proceedings of the National Academy of Sciences of the United States of America·S KochanekC T Caskey
Sep 1, 1996·Cancer Metastasis Reviews·R G Vile, H Chong

❮ Previous
Next ❯

Citations

Jul 27, 2001·Molecular Aspects of Medicine·G M Rubanyi
Oct 6, 1999·Current Opinion in Biotechnology·K W Peng, S J Russell
Sep 28, 1999·Molecular Medicine Today·K W Peng
Jun 6, 2003·Best Practice & Research. Clinical Endocrinology & Metabolism·Geoffrey Goldspink
May 11, 2013·Advances in Pharmacological Sciences·Christian AgyareMartin Adarkwa-Yiadom
Oct 26, 2013·Evidence-based Complementary and Alternative Medicine : ECAM·Christian AgyareKwesi Boadu Mensah
Jan 1, 2012·International Journal of Medicinal Chemistry·Md IrshadM Moshahid A Rizvi
Nov 17, 1999·Journal of Pharmaceutical Sciences·J A ReddyP S Low
May 19, 2009·Advanced Drug Delivery Reviews·Memy H HassanAyman Al-Hendy
Sep 7, 2006·The Journal of Gene Medicine·Daniel A WaterkampJürgen A Kleinschmidt
Feb 27, 2003·Journal of Liposome Research·A FahrSabine Brüsselbach
Sep 17, 2002·Molecular Therapy : the Journal of the American Society of Gene Therapy·Brian G BarnettJoanne T Douglas
May 7, 2002·Molecular Therapy : the Journal of the American Society of Gene Therapy·Harris SoiferNoriyuki Kasahara
Feb 19, 2000·Gene Therapy·R G VileN R Lemoine

❮ Previous
Next ❯

Related Concepts

Related Feeds

Bioinformatics in Biomedicine

Bioinformatics in biomedicine incorporates computer science, biology, chemistry, medicine, mathematics and statistics. Discover the latest research on bioinformatics in biomedicine here.

CREs: Gene & Cell Therapy

Gene and cell therapy advances have shown promising outcomes for several diseases. The role of cis-regulatory elements (CREs) is crucial in the design of gene therapy vectors. Here is the latest research on CREs in gene and cell therapy.

Related Papers

European Journal of Obstetrics, Gynecology, and Reproductive Biology
Henri MarretFlorence Fourquet
Journal of Evaluation in Clinical Practice
D A Asch, J C Hershey
BMJ : British Medical Journal
A A MelcherR G Vile
© 2022 Meta ULC. All rights reserved