article
Scientists find new agent to fight genetic disorders -- Zorro-Locked Nucleic Acid
April 30th, 2007
Contact: Cody Mooneyhan
cmooneyhan@faseb.org
301-634-7104
Federation of American Societies for Experimental Biology
A study to appear in the June 2007 issue of The FASEB Journal
describes a new agent, called "Zorro-LNA," which has the potential to
stop genetic disorders in their tracks. In the study, researchers from
the Karolinska Institute in Stockholm, Sweden, describe how they
developed Zorro-LNA to bind with both strands of a gene's DNA
simultaneously, effectively disabling that gene. This development has
clinical implications for virtually every human condition caused by or
worsened by dominant defective genes. Examples include: Huntington's
disease, familial high cholesterol, polycystic kidney disease, some
instances of glaucoma and colorectal cancer, and neurofibromatosis,
among others.
"Zorro-LNA is a new substance that targets DNA and turns off genes,"
said co-author Edvard Smith of the Karolinska Institute in Sweden. "It
has the potential of becoming a new drug for the treatment of human
genetic disease."
The findings described in this article significantly raise the
possibility that new therapies could arise where defective DNA is
deactivated more completely and more thoroughly than ever before. For
instance, Zorro-LNA could be used in combination with "RNA
interference" (RNAi). Like Zorro-LNA, RNAi has the ability to
deactivate genes, but does so by degrading the gene's RNA. In
addition, Zorro-LNA could be used to deactivate certain genes in stem
cells, which could eventually lead to the development of new cells,
tissues, or organs. The discovery of RNAi was recognized by a Nobel
Prize award in 2006 to two American scientists.
"This is a major development in the treatment not only of genetic
diseases, but also of acquired diseases when microbes or toxins cause
genes to go awry" said Gerald Weissmann, M.D., Editor-in-Chief of The
FASEB Journal. "One might say these researchers have found a gene-
hunter's Holy Grail for which scientists have been hunting for many
years. Zorro-LNA should give us a new, safe way of blocking the
effects of errors in our genetic repertoire."
###
The Editor-in-Chief of The FASEB Journal will be available for
interviews about this or other articles at a press reception at this
year's Experimental Biology meeting in Washington, D.C., on Tuesday,
May 1, 2007, between 1:30-3:00 PM in the Experimental Biology press
lounge. (Refreshments will be provided.) For more information about
the meeting, visit www.eb2007.org.
A fact sheet on this article is available at The FASEB Journal's press
room. Visit www.fasebj.org and click "Press Room" in the left column.
The FASEB Journal is published by the Federation of American Societies
for Experimental Biology (FASEB) and is consistently ranked among the
top three biology journals worldwide by the Institute for Scientific
Information.
FASEB comprises 21 nonprofit societies with more than 80,000 members,
making it the largest coalition of biomedical research associations in
the United States. FASEB advances biological science through
collaborative advocacy for research policies that promote scientific
progress and education and lead to improvements in human health.
April 30th, 2007
Contact: Cody Mooneyhan
cmooneyhan@faseb.org
301-634-7104
Federation of American Societies for Experimental Biology
A study to appear in the June 2007 issue of The FASEB Journal
describes a new agent, called "Zorro-LNA," which has the potential to
stop genetic disorders in their tracks. In the study, researchers from
the Karolinska Institute in Stockholm, Sweden, describe how they
developed Zorro-LNA to bind with both strands of a gene's DNA
simultaneously, effectively disabling that gene. This development has
clinical implications for virtually every human condition caused by or
worsened by dominant defective genes. Examples include: Huntington's
disease, familial high cholesterol, polycystic kidney disease, some
instances of glaucoma and colorectal cancer, and neurofibromatosis,
among others.
"Zorro-LNA is a new substance that targets DNA and turns off genes,"
said co-author Edvard Smith of the Karolinska Institute in Sweden. "It
has the potential of becoming a new drug for the treatment of human
genetic disease."
The findings described in this article significantly raise the
possibility that new therapies could arise where defective DNA is
deactivated more completely and more thoroughly than ever before. For
instance, Zorro-LNA could be used in combination with "RNA
interference" (RNAi). Like Zorro-LNA, RNAi has the ability to
deactivate genes, but does so by degrading the gene's RNA. In
addition, Zorro-LNA could be used to deactivate certain genes in stem
cells, which could eventually lead to the development of new cells,
tissues, or organs. The discovery of RNAi was recognized by a Nobel
Prize award in 2006 to two American scientists.
"This is a major development in the treatment not only of genetic
diseases, but also of acquired diseases when microbes or toxins cause
genes to go awry" said Gerald Weissmann, M.D., Editor-in-Chief of The
FASEB Journal. "One might say these researchers have found a gene-
hunter's Holy Grail for which scientists have been hunting for many
years. Zorro-LNA should give us a new, safe way of blocking the
effects of errors in our genetic repertoire."
###
The Editor-in-Chief of The FASEB Journal will be available for
interviews about this or other articles at a press reception at this
year's Experimental Biology meeting in Washington, D.C., on Tuesday,
May 1, 2007, between 1:30-3:00 PM in the Experimental Biology press
lounge. (Refreshments will be provided.) For more information about
the meeting, visit www.eb2007.org.
A fact sheet on this article is available at The FASEB Journal's press
room. Visit www.fasebj.org and click "Press Room" in the left column.
The FASEB Journal is published by the Federation of American Societies
for Experimental Biology (FASEB) and is consistently ranked among the
top three biology journals worldwide by the Institute for Scientific
Information.
FASEB comprises 21 nonprofit societies with more than 80,000 members,
making it the largest coalition of biomedical research associations in
the United States. FASEB advances biological science through
collaborative advocacy for research policies that promote scientific
progress and education and lead to improvements in human health.