Hutchinson-Gilford
progeria syndrome is a genetic condition characterized by the
dramatic, rapid, appearance of aging from the childhood.
Hutchinson-Gilford progeria syndrome is caused by a mutation in the
lamin A (LMNA) gene. The affected children develop a characteristic
facial experience including prominent eyes, small chin, protruding
ears, thin lips and a thin nose with a beaked tip. This syndrome also
causes hair loss (alopecia), joint abnormalities, aged-looking skin,
and a loss of fat under the skin (subcutaneous fat). Moreover,
patients of Hutchinson-Gilford progeria syndrome experiences severe
hardening of the arteries (arteriosclerosis) from the childhood. The
condition worsens with age and increases the risk of heart attack or
stroke even at a young age.
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Hutchinson-Gilford
progeria is a rare condition that affects about one in four million
newborns worldwide according to the National Institutes of Health
(NIH). Until now more than 130 cases have been reported as per the
NIH statistics. The affected patients live up to 30 years maximum,
with an average life span of 13 years. Nearly 90% of the patients die
from complications related to atherosclerosis. Till 2012 there wasn’t
any effective treatment therapy discovered for Hutchinson-Gilford
progeria syndrome. The treatments available focused mostly on
reducing cardiovascular symptoms and growth abnormalities.
Farnesyltransferase
Inhibitor (FTI)
In
2012, findings of the first clinical trial of the drug Lonafarnib, a
farnesyltransferase inhibitor (FTI), gave a new hope for the
treatment of children with Hutchinson-Gilford progeria syndrome.
Clinical trial results demonstrated improvement in weight gain,
increase in bone mineral density, reduced vascular stiffness, and
improved sensorineural hearing in patients with progeria. Previous
treatments with growth hormone, and Sulforaphane helped in reducing
the symptoms, and prolong a child’s life. However, it is essential
that the patient regularly visits the cardiologist. Rapamycin is one
other drug used before, that demonstrated to reverse nuclear
blebbing, retard cellular senescence, and facilitate degradation of
progerin.
Recently
in 2015, the scientists at the Agency for Science, Technology &
Research (A*STAR) successfully established a model of
Hutchinson-Gilford progeria syndrome. The study conducted by this
organization proposed a model which implies that progerin is linked
to telomeres. Progerin induces a reduction in heterochromatin, a
tightly packed form of DNA, making telomeres in the cell more fragile
and susceptible to damage. The damaged telomeres in turn trigger
premature cellular aging. This model is radically different from the
one believed earlier – the gene progerin caused the nucleus to be
deformed, thereby weakening the ability of cells to divide and
proliferate. The altered progerin protein makes the nuclear envelope
unstable and progressively damages the nucleus, making cells more
likely to die prematurely.
Researchers
are working to determine how genetic changes further leading to the
characteristic features of Hutchinson-Gilford progeria syndrome.
Continuous advancement in research with increased understanding of
the human aging will provide valuable insights and aid in the
treatment of this disease.
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