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Non-viral Gene Therapy - Gene Design and Delivery (Paperback, 2005 ed.)
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Non-viral Gene Therapy - Gene Design and Delivery (Paperback, 2005 ed.)
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Several years ago, when the discovery of catalytic RNA was
recognized in a public manner,many people asked if new ?elds of
therapy would soon be available. Although some tentative positive
answers were given,nobody would say with certainty that RNA of
various kinds was a truly promising means of altering gene
expression. In fact,over the past decade,both our knowledge of RNAs
with different functions and the utility of RNA in the inhibition
or enhancement of gene expression have occurred with great drama.
We proceeded in terms of possible therapeutic tools from RNase P
and group I introns through "hammerhead" RNA enzymes, antisense
technology, and more recently, to RNAi and its derivatives. A
useful practical method of RNA delivery in animals will complete
the picture. The diversity of RNA and the varied role of it inside
cells and in therapy should be a tremendous challenge for young
molecular biologists. This volume will make their task easier.
Sidney Altman Sterling Professor of Molecular,Cellular & Devel-
mental Biology,Nobel Laureate Department of Molecular,Cellular and
Developm- tal Biology Yale University V NGTPR 4/23/05 1:00 PM Page
VI VI Foreword Delivery of nucleic acids to cells in an animal
remains a challenging problem. It is the major obstacle to success
of therapeutic approaches using genes and oli-
nucleotides,including siRNAs. Solutions found so far by chemists
are satisfactory only for transfection of cells in culture.
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