Ex vivo delivery

Another vehicle for the ex vivo delivery of ribozyme genes is cationic lipids (Hope et al., 1998). Since there are a variety of formulations for these lipids, it is usually best to test a panel of lipids for those that provide the highest efficiency of gene transfer with the least toxicity. 

Vein-graft stenosis, infrainguinal bypass surgery Pressure ex vivo delivery E2F Decoy Oligonucleotide Mann, M.J., Dzau, V.J. etal. Multicenter trial, USA... 

There are two types of delivery methods in human gene therapy. The first, called ex vivo, is the type used to combat SCID. Ex vivo delivery means that somatic cells are removed from the patient, altered with the gene therapy, and then given back to... 

A comparative study on the most promising dehvery techniques is presently being undertaken in our laboratory. Based on our experience and the prehminary results from this study, we recommend a protocol based on DNA co-transfection to be the first choice for a delivery protocol ex vivo. 

Since the design of the first targeted polyplexes more than 20 years ago [97, 134], numerous efforts have been made to develop polyplexes for use in medical products, both in pharmacological animal studies and in human studies. Therapeutic modalities include ex vivo treatment of isolated human patient cells, localized in vivo treatments, and - currently the most challenging delivery scenario - in vivo targeted intravenous delivery. 

Gene Therapies. The types of vectors that have been used or proposed for gene transduction include retrovirus, adenovirus, adeno-associated viruses, other viruses (e.g., herpes, vaccinia, etc.), and plasmid DNA. Methods for gene introduction include ex vivo replacement, drug delivery, marker studies, and others and in vivo, viral vectors, plasmid vectors, and vector producer cells. 

G. M. Barratt, C. Morin, M. Appel, and I. Seyler, Intracellular delivery of a muramyl dipeptide derivative by poly(DL-lactide) nanocapsules, in In vitro and ex vivo test systems to rationalize drug design and delivery, Paris, 1993, pp. 265-268. 

S. T. K. Narishetty and R. Panchagnula. Transdermal delivery system for zidovudine In vitro, ex vivo and in vivo evaluation. Biopharm. Drug Dispos. 25 9-20 (2004). 

Keywords Nasal drug delivery Preclinical drug development In situ Ex vivo, Drug metabolism Drug transport... 

Specific Applications of Ex Vivo Models in Nasal Drug Delivery Studies... 

Sakagami M (2006) In vivo, in vitro and ex vivo models to assess pulmonary absorption and disposition of inhaled therapeutics for systemic delivery. Adv Drug Deliv Rev 58 1030-1060. 

Birchall, J. C., et al.. Gene expression in an intact ex-vivo skin tissue model following percutaneous delivery of cationic liposome-plasmid DNA complexes. Int. J. Pharm., 197, 233-38, 2000. 

Theoretically, mutated or nonfunctional genes could be excised and replaced, and new genes with desired functions could be permanently inserted into the genome. Stable integration of an antisense DNA might also be desirable in some circumstances. Because of the technical difficulties associated with the delivery of nucleic acid-based products selectively to specific target cells in vivo, more experimental information is available for ex vivo human gene therapy. 

The in vivo approach overcomes some of these limitations because a single dosage form may be applicable to a large population of patients. Delivery in vivo, however, is fraught with considerably greater complexities and challenges than those encountered in the ex vivo approach. The in vivo approach must achieve delivery in the... 

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