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Gene Therapy Applications Of RNA Interference, Presented At The ASGT 9th Annual Meeting
A study demonstrating one of the
first therapeutic applications to exploit microRNA for the treatment of
diseases such as hemophilia will be presented on Saturday, June 3 at the
9th Annual Meeting of the American Society of Gene Therapy (ASGT) in
Baltimore.
Gene therapy has been successful for certain immunodeficiencies, but
for the treatment of diseases where the patients have a functioning immune
system, such as hemophilia, it has been more difficult. The immune system
has often prohibited the gene from working because it sees it as an invader
and fights it off.
A team of scientists led by Luigi Naldini, MD, PhD, and Brian D. Brown,
PhD, at the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) in
Milan have developed a way to prevent the immune system from disturbing the
efforts of gene therapists.
To accomplish this, they utilized a newly uncovered network of genes
regulated by molecules known as microRNAs. Over the past 5 years, research
has revealed that microRNAs have a role in almost every cellular process,
including those involved in the development of cancer.
MicroRNAs downregulate the expression of specific genes in cells where
the gene is not needed, and thereby have an important influence over the
identity of a cell.
The HSR-TIGET group reasoned that they could use this natural function
of microRNA to selectively turn off the identity of their therapeutic gene
in cells of the immune system and prevent the gene from being found and
destroyed. The researchers injected mice with the gene containing an
immune- cell microRNA target sequence, and spectacularly, the mice did not
reject the gene, as previously occurred when vectors without the microRNA
target sequence were used.
"The discovery of microRNAs has changed our understanding of biology,"
Dr. Brown noted. "Almost every week a new study comes out implicating them
in some cellular process or pathology. Now we can take advantage of this
information for creating therapies."
Dr. Naldini's group is now beginning to exploit microRNAs further for
developing gene therapies to treat diseases, including hemophilia, and even
cancer.
RNAi has enormous therapeutic potential for HBV in mice
A study investigating the effects of long-term high-level expression of
short hairpin RNAs (shRNAs) in the livers of adult mice to target hepatitis
B virus (HBV), and other numerous genes, was presented today at the 9th
Annual Meeting of the American Society of Gene Therapy (ASGT) in Baltimore.
Dirk Grimm, PhD, and Mark A. Kay, MD, PhD, from Stanford University
Medical School and colleagues developed a genetically optimized shRNA
delivery vehicle based on a non-disease causing monkey virus
(Adeno-associated virus type 8) to efficiently induce RNAi in the livers of
the mice.
When infused, the vehicle infected the entire liver and resulted in
high- level shRNA expression in each liver cell.
The team discovered a limit of shRNA molecules which is tolerated by
individual cells, and that exceeding this limit often results in toxicity
and cell death. Cell death frequently occurred in 50% of all shRNAs, severe
enough to cause massive liver injury and death of the animal. In many other
cases, the mice fully recovered, but completely lost the RNAi effect.
This research strongly implies that for future clinical RNAi use in
humans, control over intra-cellular shRNAs expression levels will be
mandatory to bypass the inherent risks of this promising new technology.
Persistent and efficient silencing of HBV in transgenic mice, achieved by
Kay's team using one of their shRNAs at a low dose, demonstrates that RNAi
has, in fact, an enormous therapeutic potential.
siRNAs may be useful for reducing RSV infection
A study reporting that small inferring RNAs (siRNAs) may be useful for
inhibiting Respiratory Syncytial Virus (RSV) infection of respiratory
tissue by targeting the destruction of viral messenger RNAs (mRNAs) will be
presented on Saturday, June 3 at the 9th Annual Meeting of the American
Society of Gene Therapy (ASGT) in Baltimore.
Respiratory Syncytial Virus is a leading cause of infant
hospitalizations worldwide. Currently there is no vaccine for RSV and
treatment of acute infections is limited to supportive measures. Recent
reports also state that RSV is an important disease in elderly and
immunocompromised populations.
Anthony J. Fischer, BS, from the University of Iowa and colleagues
evaluated siRNA-based approaches to slow down viral replication in vitro
and in vivo.
The group evaluated a series of siRNAs to target a strain of RSV. They
observed a dose-dependant reduction in viral gene expression following the
administration of siRNAs into a cell culture.
This shows that siRNAs may be useful for reducing RSV infection of
respiratory tissue by destroying viral mRNAs.
Study shows useful method in treating neurodegenerative diseases
A study demonstrating a method of protein delivery to the brain may
prove useful in treating many neurodegenerative diseases, including
Alzheimer's, Parkinson's or Huntington's diseases, was presented today at
the 9th Annual Meeting of the American Society of Gene Therapy (ASGT) in
Baltimore.
Treatment of many neuronal degenerative diseases is not possible due to
the blood-brain barrier that prevents passage of most proteins and
molecules in the blood from reaching the highly fragile and protected
environment of the neurons of the brain.
Proteins that are required by the brain have specific receptors on the
blood-brain barrier that allow their transport to the neurons from the
blood.
Brian J. Spencer, PhD, and Inder M. Verma, PhD, at The Salk Institute
for Biological Studies in California hypothesized that by piggy backing on
these receptors, they could deliver a therapeutic protein to the brain to
treat a neuronal degenerative disorder.
The system was tested in a mouse model of Sly disease, a lysosomal
storage disease that leads to progressive neuronal degeneration and
eventually an early death.
Researchers found significant quantities of the fusion protein in the
brains of these mice. Improvements in coordination and balance as well as
learning and memory in these mice were also observed.
This study suggests that this fusion protein was able to prevent the
death of neurons in the mice affected with Sly disease, and that this may
be a useful method of protein delivery in the brain for other
neurodegenerative diseases.
The American Society of Gene Therapy is a professional non-profit
medical and scientific organization dedicated to the understanding,
development and application of gene and related cell and nucleic acid
therapies and the promotion of professional and public education in the
field. For more information, visit http://www.asgt.org.
American Society of Gene Therapy
http://www.asgt.org
Terapia geneticã aplicatii ale ARN Interferenta, prezentate la reuniunea anualã ASGT 9a - Gene Therapy Applications Of RNA Interference, Presented At The ASGT 9th Annual Meeting - articole medicale engleza - startsanatate