Scientists have developed a new RNA repair technology that can help fix defects in RNA that cause diseases. The technology involves identifying compounds that target expanded triplet repeats, which are sequences of three nucleotides repeated many more times than normal that are associated with neurological disorders. Researchers also describe methods for finding compounds that can repair defective RNAs. Additionally, small RNA-based drugs represent a promising approach for treating diseases by silencing genes, and researchers have discovered new mechanisms for RNA-based control of gene expression. Proper understanding and manipulation of RNA processing could lead to drugs that fix genetic mutations and reduce diseases.

1. New RNA Repair Technology and
Therapeutic Strategies for
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Controlling Gene Expression
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Anny Carolina Sarria Mena
Medicine Student, 3rd semester
Teacher: Lina María Martinez Sánchez
Molecular Biology
UPB - 2012

4. INTRODUCTION
The proper use of the tools offered
by molecular biology would be of
great benefit to modern medicine, as with so
many technological advances, to the heart of
mutations and would reduce the
pathologies that they can be repaired.
Interestingly travésde as identifyingRN
A sequences and errors in the
maturation of the
same drugs can create and repair mec
hanisms such as RNA.

5. Scientists Create Novel
RNA Repair Technology

6. Scientists Create Novel
RNA Repair Technology
 Identification a compound that can help repair a specific
type of defect in RNA, a type of genetic material.
 Methods in the new study could accelerate the
development of therapeutics to treat a variety of
incurable diseases such as Huntington's disease,
Spinocerebellar ataxia, and Kennedy disease.

7. Scientists Create Novel
RNA Repair Technology
 Describe a method to find compounds that target
defective RNAs, specifically RNA that carries a
structural motif known as an "expanded triplet repeat.
 The triplet repeat, a series of three nucleotides
repeated many more times than normal in the genetic
code of affected individuals, has been associated with
a variety of neurological and neuromuscular disorders.

8. Scientists Create Novel
RNA Repair Technology
 It has been shown recently that both the protein and
the RNA are toxic. The toxic RNA defect actually
sucks up other proteins that play critical roles in RNA
processing, and that is what contributes to these
various diseases.
 Also opens up new avenues for therapeutic
development because we have clearly demonstrated
that small molecules can reverse this type of defect.

9. COMMENT

10. New RNA-Based Therapeutic
Strategies for Controlling Gene
Expression

11. New RNA-Based Therapeutic
Strategies for Controlling Gene
Expression
 Small RNA-based nucleic acid drugs represent a
promising new class of therapeutic agents for silencing
abnormal or overactive disease-causing genes.
 Short strands of nucleic acids, called small RNAs, can
be used for targeted gene silencing, making them
attractive drug candidates.

12. New RNA-Based Therapeutic
Strategies for Controlling Gene
Expression
 Researchers have discovered new mechanisms
by which RNA drugs can control gene activity. A
comprehensive review article in Nucleic Acid
Therapeutics, a peer-reviewed journal published
by Mary Ann Liebert, Inc., details these advances.

13. New RNA-Based Therapeutic
Strategies for Controlling Gene
Expression
 Review common features shared by RNAi pathways for
controlling gene expression and focus in detail on the
potential for Argonaute-RNA complexes in gene
regulation and other exciting new options for targeting
emerging forms of non-coding RNAs and pre-mRNAs in
the article "Argonaute and the Nuclear RNAs: New
Pathways for RNA Mediated Control of Gene
Expression."

14. COMMENT

15. Medical Utility

16. Medical Utility
The RNA may represent one of the
great advances in medicine, if
used properly and that the viruses that
plague humanity are involved
in transcription and maturation of the
same.
And with the proper identification of
the processing and maturation of these
viruses can find a solution by
using drugs that can attack in its
genome.

17. Medical Utility
If accurately elucidate the general
maturation
and processing of RNA from
this may create drugs that can
"manipulate" the RNA and fix all
the mutations that can occur.

18. Medical Utility
Through the years and with the
expertise of researchers
have made ​significant progress in the
field of medicine have been
established treatments and therapies,
drugs and instruments which act
on the genome
and polymorphisms reduce all thanks
to Molecular Biology.

19. BIBLIOGRAPHY
• “Scientists Create Novel RNA Repair
Technology”, Science Daily Journal, internet
edition, January 2012,
http://www.sciencedaily.com/releases/2012/01/1
20117191544.htm
• “New RNA-Based Therapeutic Strategies for
Controlling Gene Expression”, Science Daily
Journal, internet edition, February 2012,
http://www.sciencedaily.com/releases/2012/02/1
20202164825.htm