Martin,
Thanks for posting.
Very reasonable piece, probably because it's written by expert in reasonable journal and asks for money for public research, not to buy stock. <g>
Kmiec's paper in Science in 1996 was an event...
Unfortunately, I can't get Full Text Science for 1996 on line, so have to rely on my memory (2.5 years, hmm...) and later publications
...recombination frequency three to six orders of
magnitude higher than that normally seen in cultured mammalian cells.
Huge potential, if...
Well, to make long story short:
Original paper was published in Sep 1996 in Science.
In 1997 two letters appeared as a response for the publication. They were critical. They asked for controls, controls and controls. One of the possible explanation they mentioned was contamination by wild type cells.
After reading paper and letters and respond together I found all publications reasonable, and made extremely important conclusion: "more experiments will be necessary to..."<g>.
If someone would like to read something from critics, here is free link, (read to the end):
hepatology.aasldjournals.org
Would someone decide to go deep into it, that's the list to start from (courtesy of Science magazine):
Correction of the Mutation
Responsible for Sickle Cell Anemia by
an RNA-DNA Oligonucleotide
Allyson Cole-Strauss, * Kyonggeun Yoon, * Yufei Xiang,
Bruce C. Byrne, Michael C. Rice, Jeff Gryn,
William K. Holloman, Eric B. Kmiec
A chimeric oligonucleotide composed of DNA and modified
RNA residues was used to direct correction of the mutation in
the hemoglobin S allele. After introduction of the chimeric molecule into lymphoblastoid cells
homozygous for the S mutation, there was a detectable level of gene conversion of the mutant allele to the
normal sequence. The efficient and specific conversion directed by chimeric molecules may hold promise as
a therapeutic method for the treatment of genetic diseases.
This article has been cited by other articles:
Schuster, M. J., Wu, G. Y. (1998). CHIMERIC OLIGONUCLEOTIDES: AN EXCITING ANSWER THAT
RAISES MORE QUESTIONS. Hepatology 28: 594-596
Bandyopadhyay, P., Ma, X., Linehan-Stieers, C., Kren, B. T., Steer, C. J. (1999). Nucleotide Exchange in
Genomic DNA of Rat Hepatocytes Using RNA/DNA Oligonucleotides. TARGETED DELIVERY OF
LIPOSOMES AND POLYETHYLENEIMINE TO THE ASIALOGLYCOPROTEIN RECEPTOR. J. Biol.
Chem. 274: 10163-10172
Gorman, L., Suter, D., Emerick, V., Schümperli, D., Kole, R. (1998). Stable alteration of pre-mRNA
splicing patterns by modified U7 small nuclear RNAs. Proc. Natl. Acad. Sci. U. S. A. 95: 4929-4934
Related articles in Science:
Science 1997 275: 1401-1404. (in Letters)
Sickle Cell Anemia Research and a Recombinant DNA Technique.
Andrzej Stasiak, Stephen C. West, Edward H. Egelman;, and Eric B. Kmiec;
Science 1997 277: 459-463. (in Letters)
Recombinant DNA Technique and Sickle Cell Anemia Research .
Kirk R. Thomas, Mario R. Capecchi;, and Eric B. Kmiec
Science 1997 275: 1401-1404. (in Letters)
I should have all these materials somewhere, so, if someone is in desperate need, you may contact me <g>.
About general conclusions, yes, correction would be much better solution than new, "normal", gene copy supply ... if any of these solutions were available TODAY... |
