NanoScript (left) for the regulation of DNA transcription and MitoScript (right) for the regulation of mDNA transcription.
Invention Summary:
As of 2022, 1 in 4000 people are affected by mitochondrial disorders. Currently there are no cures for many mitochondrial disorders and treatment plans typically only address alleviating symptoms. Synthetic transcription factors have shown promise in the treatment of mitochondrial disorders. However, they have faced significant challenges to their efficacy as a treatment modality; such as poor membrane penetration, intracellular degradation, low gene activation.
Rutgers researchers have developed a novel nanoparticle-based platform for synthetic transcription factors, capable of modulating endogenous DNA in a safe, non-viral fashion. NanoScript offers a modular, synthetic platform that allows for highly tunable functionality of DNA-binding domain, nuclear localization signal domain, and gene-modulation domain. MitoScript builds upon this nanoparticle technology to offer efficient, non-viral regulation to mitochondrial DNA (mtDNA) transcription. MitoScript can regulate mtDNA transcription in human cell lines selectively and offers a greater understanding of the fundamental mechanisms of mitochondrial disorders. This technology platform can assist in developing effective treatments for mitochondrial and vascular diseases.
Associated technologies:
1. 2013-013
2. 2023-105
Market Applications:
1. Research tool for cellular gene modulation
2. Gene therapy agent for:
Advantages:
Publications:
1. Yang L, Lee K-B. Nano Letter 2023 Mar 8;23(5):2046-2055. https://doi.org/10.1021/acs.nanolett.2c03958
2. Patel S., Lee K-B. ACS Nano 2014 Aug 18;8(9):8959-8967. https://doi.org/10.1021/nn501589f
Intellectual Property & Development Status:
Issued: US Patents 10,100,332 and 11,306,326 B2. Published: WO 2019/088772 (KR); US 2021/0369653. Patent pending for some content. For any business development and other collaborative partnerships contact:marketingbd@research.rutgers.edu
