Oligonucleotide therapy to help cocaine use disorder reach the brain
Official title: Oligonucleotide delivery to treat cocaine use disorder
Funded at Icahn School of Medicine at Mount Sinai · NIH-11429935 · Data updated 2026-11-12
A new ASO-based approach aims to deliver gene-targeting therapy across the blood–brain barrier to help people with cocaine use disorder.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Icahn School of Medicine at Mount Sinai NIH-funded |
| Lab location | 1 site (New York, United States) |
| Project start | August 1, 2026 |
| Project end | May 31, 2031 |
| Funding year | 2026 |
| Award (this year) | $720,691 |
| NIH project number | 1R01DA066665-01 |
| Project ID | NIH-11429935 on NIH RePORTER |
Who could benefit from this research
✓Could help
Adults with cocaine use disorder who could participate in future translational or clinical studies.
✕Less likely to help
Individuals without cocaine use disorder or those not eligible for CNS-delivery trials may not benefit.
What this research studies
This project plans to design blood–brain barrier–crossing conjugates that carry antisense oligonucleotides into the brain to modulate gene expression related to cocaine addiction. The work combines recent ASO technology with targeted delivery strategies and uses integrative transcriptomics from human and mouse models to identify transcription factors involved in addiction-related brain changes. If successful, the approach could offer a novel brain-delivery platform for treating CNS disorders where current medicines are lacking.
Why it matters
Potential benefit: If successful, patients with cocaine use disorder could gain a new, brain-targeted treatment option.
How similar studies have gone: Previous work shows promise for ASO delivery to the CNS, but effective human brain delivery remains a major challenge and this approach is innovative.
Where this research is happening
-
Icahn School of Medicine at Mount Sinai
New York, United States Active
Researchers
- Principal investigator: Dong, Yizhou — Icahn School of Medicine at Mount Sinai
- Study coordinator: Dong, Yizhou
Related research
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-
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Psilocybin-Assisted ACT for Co-Occurring Cocaine Use Disorder and Major Depressive Disorder
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Accelerated TMS for Cocaine Use Disorder
A new, faster brain-stimulation treatment is being tested to help adults with cocaine use disorder reduce cravings and use.
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Focused ultrasound for reducing cocaine craving and use
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.