The development of improved cationic lipids for gene transfer into cystic fibrosis airway epithelial cells

Article abstract of DOI:10.1080/10426509608545147

In an effort to improve the efficiency of cationic lipid mediated gene transfer, over 90 novel cationic lipids of diverse structural types were synthesized and evaluated in vitro. Four cationic lipids derived from phospholipids were examined. The most promising cationic lipid formulations were tested in vivo by intranasal or transtracheal instillation into the lungs of BALB/c mice. The most active formulations gave CAT reporter gene expression levels which are greater than 500 fold over that which could be attained using free DNA alone. Certain cationic lipid formulations have been shown to facilitate substantial expression of the CFTR (cystic fibrosis transmembrane conductance regulator) gene in vitro as determined by the SPQ and Ussing Chamber assays.

Full text of DOI:10.1080/10426509608545147

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Phosphorus, Sulfur, and Silicon and
the Related Elements
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The Development of Improved
Cationic Lipids for Gene Transfer
into Cystic Fibrosis Airway
Epithelial Cells
D. J. Harris , J. Marshall , E. R. Lee , C. S. Siegel , D.
Mcneilly , N. S. Yew , J. Nietupski , M. Nichols , M.
Cherry , N. Wan , C. Jiang , M. Lane , E. Rowe , R. K.
Scheule , A. E. Smith & S. H. Cheng
a Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
b Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
c Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
d Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
e Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
f Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
g Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
h Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
i Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
j Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
k Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
l Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
m Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA

n Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
o Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
p Genzyme Corp. , One Kendall Square, Cambridge, MA
02139, USA
Published online: 06 Aug 2008.
To cite this article: D. J. Harris , J. Marshall , E. R. Lee , C. S. Siegel , D. Mcneilly , N. S.
Yew , J. Nietupski , M. Nichols , M. Cherry , N. Wan , C. Jiang , M. Lane , E. Rowe , R. K.
Scheule , A. E. Smith & S. H. Cheng (1996) The Development of Improved Cationic Lipids for
Gene Transfer into Cystic Fibrosis Airway Epithelial Cells, Phosphorus, Sulfur, and Silicon and
the Related Elements, 109:1-4, 289-292
To link to this article: http://dx.doi.org/10.1080/10426509608545147
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