Biopump: Autologous skin-derived micro-organ genetically engineered to provide sustained continuous secretion of therapeutic proteins

Dermatologic Therapy
Eduardo MitraniAmos Panet

Abstract

A novel approach for sustained production of therapeutic proteins is described, using genetic modification of intact autologous micro-organ tissue explants from the subject's own skin. The skin-derived micro-organ can be maintained viable ex vivo for extended periods and is transduced with a transgene encoding a desired therapeutic protein, resulting in protein-secreting micro-organ (biopump (BP)). The daily protein production from each BP is quantified, enabling drug dosing by subcutaneous implantation of the requisite number of BPs into the patient to provide continuous production to the circulation of a known amount of the therapeutic protein. Each implanted BP remains localized and is accessible, to enable removal or ablation if needed. Examples from preclinical and clinical studies are presented, including use of associated virus vector 1 and helper-dependent adenoviral vectors producing BPs to provide long-term sustained secretion of recombinant interferon-α and erythropoietin.

References

Aug 1, 1989·The Journal of Investigative Dermatology·K S StennE Kuklinska
Sep 1, 1985·Cell Differentiation·G R CunhaY Sugimura
Oct 27, 1999·Proceedings of the National Academy of Sciences of the United States of America·N MorralA L Beaudet
Feb 28, 2003·Proceedings of the National Academy of Sciences of the United States of America·Yong ZhaoEliezer Huberman
Nov 6, 2003·Molecular Therapy : the Journal of the American Society of Gene Therapy·Donna Palmer, Philip Ng
Dec 8, 2004·Journal of Hepatology·Amikam GershonowitzEduardo Mitrani
Jan 4, 2005·Journal of Vascular Research·Eilat HassonEduardo Mitrani
Mar 4, 2005·The Journal of Gene Medicine·E HassonE Mitrani
Apr 7, 2005·Differentiation; Research in Biological Diversity·Eduardo MitraniYael Shimoni
Jul 27, 2005·Molecular Therapy : the Journal of the American Society of Gene Therapy·Einat Brill-AlmonNoam Shani
Jan 13, 2006·Journal of Vascular Research·Eilat HassonEduardo Mitrani
Oct 30, 2007·American Journal of Physiology. Heart and Circulatory Physiology·Pierre VoisineRoger J Laham
Feb 22, 2008·Human Gene Therapy·Nikolai KunicherAmos Panet
Mar 23, 2011·Human Reproduction·Ariel RevelEduardo Mitrani
May 21, 2011·Tissue Engineering. Part C, Methods·Yulia ShamisEduardo Mitrani

❮ Previous
Next ❯

Related Concepts

Related Feeds

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.