Related Experiment Video
Updated: Mar 13, 2026
06:53
A Simple Alternative to Stereotactic Injection for Brain Specific Knockdown of miRNA
Published on: December 26, 2015
13.2K
Retro-1 Analogues Differentially Affect Oligonucleotide Delivery and Toxin Trafficking.
Bing Yang1, Xin Ming1, Hajer Abdelkafi2
1Division of Molecular Pharmaceutics, UNC Eshelman School of Pharmacy, University of North Carolina, Chapel Hill, NC, 27599, USA.
Chemmedchem
|October 26, 2016
Summary
Retro-1, a small molecule, has two biological activities: blocking toxins and enhancing oligonucleotide delivery. Studies show these actions involve distinct cellular targets, not a single one.
Area of Science:
- Cell biology
- Molecular pharmacology
- Drug discovery
Background:
- Retro-1 is a small molecule with dual biological activities impacting cellular processes.
- It interferes with intracellular toxin trafficking and aids oligonucleotide release from endosomes.
Purpose of the Study:
- To investigate whether the two distinct biological activities of Retro-1 share a common cellular target.
- To differentiate the molecular mechanisms underlying Retro-1's effects on toxins and oligonucleotides.
Main Methods:
- Synthesis and testing of several Retro-1 analogues.
- Evaluation of analogue activity against specific toxins.
- Assessment of analogue efficacy in enhancing oligonucleotide delivery.
Main Results:
- Specific Retro-1 analogues were identified that modulated toxin activity independently of oligonucleotide delivery.
- Other analogues were found to enhance oligonucleotide delivery without affecting toxin activity.
- Retro-1 was the only compound tested that exhibited both biological activities.
Conclusions:
- The molecular targets responsible for Retro-1's effects on toxin trafficking and oligonucleotide release are distinct.
- This finding has implications for understanding intracellular transport pathways and developing targeted therapeutics.
Related Concept Videos
03:18Non-LTR Retrotransposons
13.8K
As the name suggests, non-LTR retrotransposons lack the long terminal repeats characteristic of the LTR retrotransposons. Additionally, both LTR and non-LTR retrotransposons use distinct mechanisms of mobilization. Non-LTR retrotransposons are further divided into two classes - Long interspersed nuclear elements (LINEs) and short interspersed nuclear elements (SINEs), both of which occur abundantly in most mammals, including humans. Some of the active non-LTR retrotransposons in humans are L1...
13.8K
01:11Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase
61
Genetic polymorphisms in drug targets have emerged as critical determinants of interindividual variability in drug response and toxicity. Pharmacogenomic investigations increasingly focus on identifying these variations to personalize and optimize therapeutic interventions. A drug target may be a receptor, enzyme, or signaling protein involved in pharmacologic responses or disease-related pathways. While early pharmacogenetic studies focused primarily on drug metabolism, current research...
61
01:24Modified-Release Drug Delivery Systems: Site-Targeted
70
Site-targeted drug delivery systems enhance therapeutic efficacy while minimizing systemic toxicity and treatment costs. Unlike conventional methods, these systems ensure precise drug delivery, improving bioavailability and reducing side effects. Targeted drug delivery is classified into three levels. First-order targeting directs drugs to the capillary beds of specific organs or tissues. Second-order targets specific cell types, such as tumor cells, using receptor-mediated interactions.
70
01:24Drug Toxicity: Dose-Dependent Reactions
99
Drug toxicities can be stratified into pharmacological, pathological, or genotoxic based on their mechanisms. The incidence and severity of these toxicities generally increase with the drug's concentration in the body and exposure time.Pharmacological toxicity is evident when the therapeutic effects of drugs overshoot into adverse reactions in a predictable, dose-dependent manner. Central nervous system (CNS) depression from barbiturates is a classic example, with effects escalating from...
99
01:29Drugs Affecting Neurotransmitter Synthesis
2.4K
Drugs affecting neurotransmitter synthesis can impact the adrenergic neuron and the synthesis of neurotransmitters. For example, α-methyltyrosine and carbidopa target specific enzymes involved in catecholamine synthesis. α-methyltyrosine inhibits the enzyme tyrosine hydroxylase, which converts tyrosine into dopamine. By blocking this enzyme, α-methyltyrosine reduces dopamine production and other catecholamines. Carbidopa, on the other hand, inhibits the enzyme dopa decarboxylase,...
2.4K
01:26Targets for Drug Action: Overview
10.9K
Drugs target macromolecules to modify ongoing cellular processes. Primary drug targets include receptors, ion channels, transporters, and enzymes.
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
Receptors are either membrane-spanning or intracellular proteins, which upon binding a ligand, get activated and transmit the signal downstream to elicit a response. Drugs bind receptors, either mimicking the action of endogenous ligands or blocking the receptor activity to bring about a modified response. Nearly 35% of approved drugs target the G...
10.9K