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Delivery of chemically modified PNA oligomers
Phone: (864) 656-1106
Phone: (864) 207-0002
Type: Nonprofit college or university
PROJECT SUMMARYRNA is a validated target for drug design, both as therapeutic and as a target.
Targeting specific RNA, such as rRNA which are involved in proliferation and survival of
bacteria is a promising approach. We are developing fast and low cost methods to
screen sequence-specific small molecules for novel anti-ribosomal activities. We will
construct sequence-specific chemically modified ribosomal targeting oligomers that can
be effectively delivered inside the cell, addressing the key objective of
PAR-17-036 (to generate new technologies and products for delivering nucleic
acids into cells and tissues for the purpose of treatment or prevention of
human disease). Complexes between ribosomal components will be exploited
as targets for small molecule drug libraries that-inactivate the ribosome.
NUBADs unique experimental approaches and technologies will allow us to target
ribosomal regions not previously explored for susceptibility against microbial
targets.The work proposed here, a multidisciplinary effort encompassing solid-phase
organic synthesis, oligonucleotide delivery, RNA targeted screening, antimicrobial
activity, and in vivo efficacy studies describes the development of sequence-specific cell
permeable binders of rRNA. The success of the proposed work would be a significant
addition to currently available ribosome-specific approaches in drug development. We
propose using a small rRNA target sequences to design conjugates that can be
employed to inhibit microbial growth, opening possibilities for developing sequence-
specific RNA targeted therapeutics.Health Relevance Statement.
The work proposed here, a multidisciplinary effort encompassing organic synthesis,
oligonucleotide (PNA) delivery, RNA targeted screening and in vivo efficacy studies,
describes the development of sequence-specific cell permeable binders of rRNA as
PNA based therapeutics. We propose using a small rRNA target sequences to design
conjugates that can be employed to inhibit microbial growth, opening possibilities for
developing sequence-specific RNA targeted therapeutics.
* Information listed above is at the time of submission. *