By Syed Akbar
Hyderabad: In a boost to the fast emerging gene therapy, city
scientists have successfully synthesized orally administered next
generation nanoparticles to deliver biomaterials like DNA and RNA to
cure certain diseases.
Scientists at the Indian Institute of Chemical Technology (IICT)
developed the orally administered nanoparticles from organic
materials. They are useful for delivery of small regulatory RNA or
siRNA and DNA as therapeutic materials. They also serve as novel
material or cargoes for molecular diagnostics, since the nanoparticles
These nanoparticles show no side effect to surrounding tissues. They
are eliminated from the body after performing their function. The IICT
scientists have successfully demonstrated both in lab and animal
models how effective delivery of specific siRNA would silence the
target gene to treat health problems related to genetics.
Dr Manika Pal Bhadra, who led the research, has been awarded National
Academy of Sciences India-Reliance Industries Platinum Jubilee Award,
says an official press release here on Thursday.
To accomplish oral delivery of siRNA adult Drosophila (fruit fly) were
fed with these nanoparticles. Nanoparticles feeding caused no
impairment of their sex, egg laying capacity or survivality proving
the non-toxic nature of the synthesised particles.
Experiments using siRNA oligonucleotides against green fluorescence
protein and red fluorescent protein gene showed a dramatic down
regulation of their product. A comparative study using commercially
available gene delivery vehicle showed a better silencing with almost
zero toxicity to the flies.
RNA interference (RNAi) is a cellular process whereby expression of
any particular gene is silenced by small RNAs. These small pieces of
RNAs also known as short interfering RNAs is capable of turning off
any gene and thereby stop the production of specific proteins, making
them a new class of therapeutic drugs.
It has significant potential for the treatment of human diseases.
Still the major barrier is the safe and effective delivery of RNA to
target tissues. The IICT research solves the problem. Several factors
play important role for siRNA delivery, including the chemical
modification of the delivery vehicle and its biological responses to
siRNA sequences and structure.
In recent years, a wide variety of inorganic nanomaterials has been
used as delivery vehicles. But two major issues i.e., targeted release
of the biomolecules and rapid clearance of the carriers that are
considered for delivery in live cells still remain unanswered. It has
led to the failure of many inorganic nanostructures as attractive
vehicles and has opened a window of opportunity for the development of
nanoparticles from organic materials that are well accepted in
bio-systems along with clearance from the live cells.
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