Small molecule targeting of RNA structures in neurological disorders.

Small molecule targeting of RNA structures in neurological disorders.

Publication date: Apr 09, 2019

Aberrant RNA structure and function operate in neurological disease progression and severity. As RNA contributes to disease pathology in a complex fashion, that is, via various mechanisms, it has become an attractive therapeutic target for small molecules and oligonucleotides. In this review, we discuss the identification of RNA structures that cause or contribute to neurological diseases as well as recent progress toward the development of small molecules that target them, including small molecule modulators of pre-mRNA splicing and RNA repeat expansions that cause microsatellite disorders such as Huntington’s disease and amyotrophic lateral sclerosis. The use of oligonucleotide-based modalities is also discussed. There are key differences between small molecule and oligonucleotide targeting of RNA. The former targets RNA structure, while the latter prefers unstructured regions. Thus, some targets will be preferentially targeted by oligonucleotides and others by small molecules.

Angelbello, A.J., Chen, J.L., and Disney, M.D. Small molecule targeting of RNA structures in neurological disorders. 06440. 2019 Ann N Y Acad Sci.

Concepts Keywords
Amyotrophic Lateral Sclerosis Antisense therapy
Huntington Molecular biology
Microsatellite Applied genetics
MRNA Therapeutic gene modulation
Neurological Oligonucleotide
Neurological Diseases Biotechnology
Neurological Disorders Diseases
Oligonucleotide Life sciences
Oligonucleotides Nucleic acids
Pathology Branches of biology
RNA Amyotrophic lateral sclerosis
Small Molecule


Type Source Name
disease DOID amyotrophic lateral sclerosis
disease MESH amyotrophic lateral sclerosis
disease MESH development
disease MESH disease progression
disease MESH neurological disorders

Original Article

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