A gradient
is a pathway of increasing concentration of a
specific substance. Overall, our findings suggest that
if this
technique could be adapted to treat children with this type of LSD we would
have an effective
therapy.,
President and Chief Executive Officer, will speak at the CIBC World
Markets Annual Biotechnology
and Specialty Pharmaceuticals Conference
on Tuesday, April 5, 2005, at 1:45 p.
society hormones
bilingual technology
Therefore, genetically
modified BMCs would be able to produce an endless
supply of monocytes carrying the gene for beta
-galactosidase, ensuring that
the enzyme would always be available to affected neurons in the brain
of
children with GM1 - gangliosidosis.
Lysosomes are the cell's recycling centers, where proteins
, fats and other
molecules are broken down into their basic building blocks, which are then
reused
to make new molecules.
The other authors of this paper include Alessandra Tessitore (currently
at
Telethon Institute of Genetics and Medicine; Naples, Italy) and Angela
Ingrassia (St. Jude
is financially supported by ALSAC, its fund-raising organization. The company has
built a diverse
portfolio of product candidates targeted to many types
of cancer, including three being tested in
multiple ongoing clinical
trials, SGN-30, SGN-15 and SGN-40, and four in preclinical
development
, SGN-35, SGN-70, SGN-75 and SGN-17/19.genetics hormones
"The improvement was clearly linked
to the ability of the
corrected neurons to break down excess GM1 with the
enzyme delivered by the monocytes.html).issues bilingual
After the St.org/media/0,2561,453_5297_12276,00.m. The conference
is being held at the Millennium
Broadway Hotel in New York.
More information about Seattle Genetics can be found at
www.com
b genetically
These cells took in the enzyme
released by the monocytes and used it to break down excess
GM1, thus
correcting the potentially fatal buildup of this molecule.
"We used the brain's
own signaling molecules to guide the genetically
modified monocytes along a concentration gradient
to the degenerating brain
cells," d'Azzo said.
d'Azzo previously showed that GM1 - gangliosidosis
is an inherited disease
in which one of the enzymes in the lysosomes is defective (Molecular Cell
,
Sept. Jude).
d'Azzo holds the Chair in Genetics and Gene Therapy endowed by the Jewelers
Fund
for Children.stjude.Seattle Genetics to Present at CIBC Biotechnology + Specialty Pharmaceuticals Conference
Seattle Genetics has
developed leading ADC technology comprised of highly potent synthetic
drugs
and stable linkers for attaching the drugs to monoclonal
antibodies.science food
"We showed that improvement
of the disease was directly related to the
amount of genetically modified monocytes reaching the
degenerating brain
cells," said Renata Sano, Ph.stjude.D.growth society
Jude study, the BMCs followed the gradient
toward increasing concentrations of the chemokines until they reached the site
of the degenerating
neurons. Founded by late entertainer Danny Thomas and based in
Memphis, Tenn.org. (Nasdaq:SGEN) announced
today that Clay B.com,
under the "news and investor information" section.education society
A report on this work appears
in the
prepublication online issue of Blood. Jude researchers successfully treated a laboratory model
of an LSD
called GM1 - gangliosidosis using bone marrow cells (BMCs) into which
scientists inserted
the gene for an enzyme that breaks down a fat molecule
called GM1. In the St.D.
St.modified b
, a member
of the St."
Key to the promise of curing LSDs in children using this technique is the
fact
that since BMCs are stem cells, they continually give rise to all the
various types of blood cells
in the body, including monocytes, d'Azzo said. These diseases, including
GM1 - gangliosidosis, are
responsible for most severe cases of
neurodegeneration and mental retardation among children. Jude
Children's Research Hospital is internationally recognized for
its pioneering work in finding cures
and saving children with cancer and other
catastrophic diseases.
About Seattle Genetics
Seattle Genetics discovers and develops monoclonal antibody-based
therapeutics to treat cancer
and other human diseases.birth bovine
But in GM1 -
gangliosidosis, brain cells lack this enzyme-beta-galactosidase
-and GM1
accumulates to such a high concentration that it disrupts the proper function
of the cell
and causes it to self-destruct.
The monocytes homed in on the brain by following a trail of signaling
molecules that were released by cells adjacent to the degenerating neurons,
according to Alessandra
d'Azzo, Ph. Under a disease condition, these brain cells, called astrocytes
and microglia, release
chemokines in order to trigger a migration of immune
cells to areas of the brain that are damaged
. If the BMCs were harvested from the same
child who is treated with them after these cells are genetically
modified,
this would eliminate the potential complications arising from transplanting
BMCs from
a donor. 10, 2004; http://www. LSDs occur when lysosomes lack the enzymes they
need to perform their
recycling tasks, leading to abnormal accumulation of the
molecules the lysosome is supposed to break
down.
This work was supported in part by the National Institutes of Health, a
Cancer Center
Support Grant, the Assisi Foundation of Memphis and ALSAC. For
more information, please visit http:
//www.
Business Editors/Health/Medical Writers
CIBC World Seattle Genetics,
Inc.planning birth
, Physicians
might one day be able
to treat a disease that destroys brain cells in children using genetically
modified cells to transport a "drug" to the site of the dying neural cells
(cells that transmit
impulses). This discovery occurred based on results of a
laboratory study of the technique published
by investigators at St. Such signaling proteins are called
chemokines.seattlegenetics.modified genetically
Jude team
infused the genetically modified BMCs into the
laboratory model, resulting monocytes migrated to
the degenerating brain cells
that lacked the gene for beta-galactosidase.D. Jude department of
Genetics
and Tumor Cell Biology. Normally, the immune cells recruited to the brain
by chemokines would cause
inflammation and worsen the damage. Jude Children's Research Hospital
St. Siegall, Ph.growth hormones
Jude researchers
MEMPHIS, Tenn. But in this
case, the genetically modified monocytes restored the beta
-galactosidase
activity, which in turn decreased the extent of neuron degeneration and
chemokine
levels. Dr.hormones society
Immune system cells find damaged neural cells by following a trail of protein
signals
released as part of inflammatory response to lysosomal storage disease
process
, according to St.
There is currently no cure for such disorders, which are called lysosomal
storage
diseases (LSDs)., a researcher in St.
St. Jude freely shares its discoveries with scientific
and
medical communities around the world. No family ever pays for treatments not
covered by insurance
, and families without insurance are never asked to pay.education genetically
Genetically Modified Cells Migrate to Brain and
Treat Neurodegeneration in St. Jude Model
Siegall will present an overview of key company programs
and discuss
recent activities.education control
GM1 is a critical component of normal brain cells. Jude Genetics and
Tumor
Cell Biology and the paper's first author., St.bovine family
Jude
Children's Research Hospital. d'Azzo
is senior author of the
article in Blood.
A webcast of the presentation will be available
live and for
replay from the Seattle Genetics website, www. The product
candidates encompass three
platform technologies: genetically
engineered monoclonal antibodies, antibody-drug conjugates (ADCs
) and
antibody-directed enzyme prodrug therapy (ADEPT). The company currently has license agreements
for its ADC
technology with Genentech, UCB Celltech, Protein Design Labs, CuraGen
and Bayer and
for its ADEPT technology with Genencor International.bovine genetics
The St. BMCs include a population of so
-
called pluripotent stem cells-cells that give rise to a variety of different
cell types that
have specific functions, such as the immune cells called
monocytes. Eastern Time.seattlegenetics
.hormones genetics
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