Targeting specific brain cells for gene delivery and reprogramming

Selective targeting of CNS cells in vivo for multiple transgene delivery and neuronal reprogramming

NIH-funded research Rosalind Franklin Univ of Medicine & Sci · NIH-11039998

This study is looking at ways to make sure that gene therapies can safely and effectively deliver genetic material to specific brain cells, like astrocytes, without affecting other types of cells, so that treatments for brain conditions can work better and more reliably.

Quick facts

Grant typeR21 grant
Study typeNIH-funded research
Funding institutionRosalind Franklin Univ of Medicine & Sci NIH-funded
Lab location1 site (North Chicago, United States)
Project IDNIH-11039998 on NIH RePORTER

What this research studies

This research focuses on improving the delivery of genetic material to specific cell types in the central nervous system (CNS) using viral vectors. The goal is to enhance the selectivity of these vectors to ensure they only infect the intended cells, such as astrocytes, while avoiding unintended infections of other cell types like neurons. By achieving this, the researchers aim to reduce variability in study outcomes and improve the effectiveness of gene therapies. This project will explore innovative methods to enable precise targeting and subsequent gene delivery to previously infected cells.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals aged 21 and older who have conditions affecting the central nervous system and are interested in novel gene therapy approaches.

Not a fit: Patients with conditions unrelated to the central nervous system or those under 21 years old may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could lead to more effective gene therapies for neurological conditions by ensuring that treatments are delivered precisely to the right cells.

How similar studies have performed: While the use of viral vectors for gene delivery is well-established, this specific approach to enhance cell-type selectivity is innovative and has not been extensively tested in prior studies.

Where this research is happening

North Chicago, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.