Progress in the molecular pathogenesis and nucleic acid therapeutics for Parkinson's disease in the precision medicine era

D Li, FL Mastaglia, S Fletcher… - Medicinal Research …, 2020 - Wiley Online Library
Parkinson's disease (PD) is one of the most common neurodegenerative disorders that
manifest various motor and nonmotor symptoms. Although currently available therapies can …

Therapeutically significant microRNAs in primary and metastatic brain malignancies

AA Balachandran, LM Larcher, S Chen, RN Veedu - Cancers, 2020 - mdpi.com
Simple Summary The overall survival of brain cancer patients remains grim, with
conventional therapies such as chemotherapy and radiotherapy only providing marginal …

Structure–Activity Relationship Study of a Potent α‐Thrombin Binding Aptamer Incorporating Hexitol Nucleotides

M De Fenza, E Eremeeva, R Troisi… - … A European Journal, 2020 - Wiley Online Library
The replacement of one or more nucleotide residues in the potent α‐thrombin‐binding
aptamer NU172 with hexitol‐based nucleotides has been devised to study the effect of these …

Development of Novel Chemically-Modified Nucleic Acid Molecules for Efficient Inhibition of Human MAPT Gene Expression

M Chakravarthy, S Chen, T Wang, RN Veedu - Genes, 2020 - mdpi.com
The hyperphosphorylation of the microtubule-associated protein tau (MAPT) has been
implicated in various neurological diseases, including Alzheimer's disease. It has been …

Novel disulfide-bridged bioresponsive antisense oligonucleotide induces efficient splice modulation in muscle myotubes in vitro

BT Le, TR Kosbar, RN Veedu - ACS omega, 2020 - ACS Publications
Splice-modulating antisense therapy has shown tremendous potential in therapeutic
development in recent years with four FDA-approved antisense drugs since 2016. However …

Alpha-l-Locked Nucleic Acid-Modified Antisense Oligonucleotides Induce Efficient Splice Modulation In Vitro

P Raguraman, T Wang, L Ma, PT Jørgensen… - International Journal of …, 2020 - mdpi.com
Alpha-l-Locked nucleic acid (α-l-LNA) is a stereoisomeric analogue of locked nucleic acid
(LNA), which possesses excellent biophysical properties and also exhibits high target …

Antisense oligonucleotide-mediated exon skipping strategies as the treatment for rare diseases

D Li - 2020 - researchportal.murdoch.edu.au
Antisense oligomer-mediated exon skipping is being developed as a strategy for
personalised treatment of genetic disorders through excising “in-frame” exons carrying small …