Seminar by Prof. Rakesh N. Veedu, Head, Precision Nucleic Acid Theranostics Group, Murdoch University, Australia on "Novel chemically-modified precision nucleic acids for disease specific therapy"
21 Jan 2019
Seminar Room #350
Talk Title : "Novel chemically-modified precision nucleic acids for disease specific therapy"
Abstract
Oligonucleotides
-
based technologies such as antisense oligonucleotides (AOs), DNAzymes
and aptamers attracted significant attention in recent years. We have recently investigated
the potential of novel chemica
lly
-
modified AOs containing
LNA, HNA, CeNA,
modified
morpholino
s
,
serinol nucleic acids
and
other
chemistries
to modulate RNA splicing in
mdx
mouse myotubes towards improving the therapy against Duchenne muscular dystrophy.
Our results showed that all AOs induced
Dmd
exon
-
23 skipping
[1
-
4]
. In another study, we
investigated the potential of DNAzymes to inhibit integrin alpha
-
4 gene transcript (
ITGA
-
4
)
towards developing a therapeutic molecule for tackling inflammation in multiple sclerosis.
Our results demonstrated that RNV143A, an arm
-
loop
-
arm type DNAzyme modified with a
3’
-
inverted dT efficiently inhibited
ITGA
-
4
RNA
[5]
. Recently, we also de
veloped a novel
aptamer
[6]
targeting
low
-
molecular weight
amyloid
-
beta peptides. Our aptamer
RNV95
efficiently detected low
-
molecular weight amyloid beta peptide aggregates in
the
brain
tissue samples of pathologically confirmed Alzheimer disease patients
, and offer a great
promise towards the diagnosis and therapy of Alzheimer disease
[7]
.
In addition, we also
developed novel m
uscle cells
, and cancer cell
s
-
specific
DNA
cargo molecules for
targeted
imaging and the
delivery of gene
-
targeting drugs to muscle
s and cancer
tissues
respectively
.