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Related Concept Videos

The Central Dogma01:25

The Central Dogma

Overview
pre-mRNA Processing02:01

pre-mRNA Processing

In eukaryotic cells, transcripts made by RNA polymerase are modified and processed before exiting the nucleus. Unprocessed RNA is called precursor mRNA or pre-mRNA to distinguish it from mature mRNA.
Once about 20-40 ribonucleotides have been joined together by RNA polymerase, a group of enzymes adds a “cap” to the 5’ end of the growing transcript. In this process, a 5’ phosphate is replaced by modified guanosine that has a methyl group attached to it (7-Methyl guanosine). This 5’ cap helps the...
tRNA Activation02:26

tRNA Activation

Aminoacyl-tRNA synthetases are present in both eukaryotes and bacteria. Though eukaryotes have 20 different aminoacyl-tRNA synthetases to couple to 20 amino acids, many bacteria do not have genes for all of these aminoacyl-tRNA synthetases. Despite this, they still use all 20 amino acids to synthesize their proteins. For instance, some bacteria do not have the gene encoding the enzyme that couples glutamine with its partner tRNA. In these organisms, one enzyme adds glutamic acid to all of the...
Nonsense-mediated mRNA Decay02:27

Nonsense-mediated mRNA Decay

The Upf proteins that carry out nonsense-mediated decay (NMD) are found in all eukaryotic organisms, including humans. Each protein has an individual role, but they need to work in collaboration. Upf1 is an ATP-dependent RNA helicase that unwinds the RNA helix. Because Upf1 can unwind any RNA, Upf2 and Upf3 are required to help Upf1 discriminate between nonsense and normal mRNAs.
Usually, Upf3 binds to an Exon Junction Complex (EJC) at mRNA splice sites. If a ribosome fully translates the mRNA,...
Leaky Scanning02:28

Leaky Scanning

During most eukaryotic translation processes, the small 40S ribosome subunit scans an mRNA from its 5' end until it encounters the first start AUG codon. The large 60S ribosomal subunit then joins the smaller one to initiate protein synthesis. The location of the translation initiation is largely determined by the nucleotides near the start codon as there may be multiple translation initiation sites present on the mRNA.  Marilyn Kozak discovered that the sequence RCCAUGG (where R stands for...
Nucleic Acid Structure01:25

Nucleic Acid Structure

The pentose sugar in DNA is deoxyribose, while in RNA the pentose sugar is ribose. The difference between the sugars is the presence of the hydroxyl group on the ribose's second carbon and a hydrogen on the deoxyribose's second carbon. The phosphate residue attaches to the hydroxyl group of the 5′ carbon of one sugar and the hydroxyl group of the 3′ carbon of the sugar of the next nucleotide, which forms  a 5′ to 3′ phosphodiester linkage.
DNA Structure
DNA has a double-helix structure. The...

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Related Experiment Video

Updated: Jun 12, 2026

A Simple Alternative to Stereotactic Injection for Brain Specific Knockdown of miRNA
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AI-Validated Brain Targeted mRNA Lipid Nanoparticles with Neuronal Tropism.

Mor Sela1, Gal Chen1,2, Haim Kadosh1,2

  • 1The Louis Family Laboratory for Targeted Drug Delivery and Personalized Medicine Technologies, Department of Chemical Engineering, Technion─Israel Institute of Technology, Haifa 3200003, Israel.

ACS Nano
|September 16, 2025
PubMed
Summary

Researchers developed acetylcholine-conjugated lipid nanoparticles (LNPs) for enhanced brain delivery. These targeted LNPs improve gene expression in the central nervous system (CNS), overcoming blood-brain barrier challenges.

Keywords:
Artificial IntelligenceBlood−Brain BarrierBrain TargetingCentral Nervous System (CNS)Gene DeliveryLipid NanoparticlesmRNA

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Area of Science:

  • Neuroscience
  • Biotechnology
  • Nanomedicine

Background:

  • The blood-brain barrier (BBB) restricts the delivery of therapeutics to the brain.
  • Developing effective strategies for brain-targeted drug and gene delivery is crucial for treating neurological disorders.

Purpose of the Study:

  • To engineer messenger RNA-loaded lipid nanoparticles (mRNA-LNPs) functionalized with small molecules to enhance brain delivery and gene expression.
  • To identify optimal small-molecule ligands for improved brain tropism and transfection efficacy.

Main Methods:

  • mRNA-LNPs were functionalized with various small molecules, including acetylcholine, and screened in vitro and in vivo.
  • An AI model was developed to predict BBB permeability of small-molecule ligands.
  • Biodistribution, transfection efficiency in neurons and astrocytes, and BBB penetration were assessed using various models, including human BBB-on-a-chip and brain organoids.

Main Results:

  • Acetylcholine-conjugated LNPs demonstrated superior brain tropism and gene expression compared to other tested modifications.
  • The AI model accurately predicted experimental BBB permeability results.
  • Acetylcholine-LNPs preferentially transfected neurons and astrocytes and successfully crossed human BBB models, enabling transgene expression.

Conclusions:

  • Acetylcholine-functionalized LNPs offer a promising strategy for targeted gene delivery across the BBB.
  • The developed framework provides a predictive and modular approach for engineering CNS-targeted LNPs for brain disorders.
  • This advancement holds potential for precision gene delivery in treating central nervous system diseases.