ArticleProceedings of the National Academy of Sciences of the United States of America2016
Targeted axonal import (TAxI) peptide delivers functional proteins into spinal cord motor neurons after peripheral administration.
Article in Proceedings of the National Academy of Sciences of the United States of America, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 17 papers.
What it found
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The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
17 citing papers in PubMed, 37 citations in OpenAlex.
- Transneuronal Transport of Ligand-Conjugated Upconversion Nanoparticles from the Peripheral to the Central Nervous System.JACS Au · 2026Article
- In Vivo Cytosolic Delivery of Biomolecules into Neurons for Super-Resolution Imaging and Genome Modification.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2025Article
- Insulin-inspired hippocampal neuron-targeting technology for protein drug delivery.Proceedings of the National Academy of Sciences of the United States of America · 2024Article
- Combinatorial design of ionizable lipid nanoparticles for muscle-selective mRNA delivery with minimized off-target effects.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- In vivo selection of synthetic nucleocapsids for tissue targeting.Proceedings of the National Academy of Sciences of the United States of America · 2023Article
- TAxI-peptide targeted Cas12a ribonuclease protein nanoformulations increase genome editing in hippocampal neurons.Journal of controlled release : official journal of the Controlled Release Society · 2023Article
- Retrograde Axonal Transport of Liposomes from Peripheral Tissue to Spinal Cord and DRGs by Optimized Phospholipid and CTB Modification.International journal of molecular sciences · 2022Article
- Drug delivery to the central nervous system.Nature reviews. Materials · 2022Article
- Review
- Old and new biomarkers for volumetric muscle loss.Current opinion in pharmacology · 2021Review
- Advances in nanotechnology-based strategies for the treatments of amyotrophic lateral sclerosis.Materials today. Bio · 2020Review
- Breaking Barriers: Bioinspired Strategies for Targeted Neuronal Delivery to the Central Nervous System.Pharmaceutics · 2020Review
- Systemically Administered, Target-Specific, Multi-Functional Therapeutic Recombinant Proteins in Regenerative Medicine.Nanomaterials (Basel, Switzerland) · 2020Review
- Targeting Ligands Deliver Model Drug Cargo into the Central Nervous System along Autonomic Neurons.ACS nano · 2019Article
- Peptide-siRNA Supramolecular Particles for Neural Cell Transfection.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2019Article
- Current state of in vivo panning technologies: Designing specificity and affinity into the future of drug targeting.Advanced drug delivery reviews · 2018Review
- Non-Viral Nucleic Acid Delivery Strategies to the Central Nervous System.Frontiers in molecular neuroscience · 2016Review
Corrections and comments
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Authors and funding
7 authors at 4 institutions in 1 country.
Funding
Abstract
A significant unmet need in treating neurodegenerative disease is effective methods for delivery of biologic drugs, such as peptides, proteins, or nucleic acids into the central nervous system (CNS). To date, there are no operative technologies for the delivery of macromolecular drugs to the CNS via peripheral administration routes. Using an in vivo phage-display screen, we identify a peptide, targeted axonal import (TAxI), that enriched recombinant bacteriophage accumulation and delivered protein cargo into spinal cord motor neurons after intramuscular injection. In animals with transected peripheral nerve roots, TAxI delivery into motor neurons after peripheral administration was inhibited, suggesting a retrograde axonal transport mechanism for delivery into the CNS. Notably, TAxI-Cre recombinase fusion proteins induced selective recombination and tdTomato-reporter expression in motor neurons after intramuscular injections. Furthermore, TAxI peptide was shown to label motor neurons in the human tissue. The demonstration of a nonviral-mediated delivery of functional proteins into the spinal cord establishes the clinical potential of this technology for minimally invasive administration of CNS-targeted therapeutics.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.