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Publications and Presentations

SCAD: A Modular Platform for Efficient Delivery of Duplex RNA to the CNS and Beyond

Li LC, Kang M, Lin WH, Xu F, Zhou X, Si C, Dai J, He J, Schacht I, Li Y, Gan Z. SCAD: A Modular Platform for Efficient Delivery of Duplex RNA to the CNS and Beyond. Research Square. March 26th, 2025. DOI: https://doi.org/10.21203/rs.3.rs-5904176/v1

RNAa: Mechanisms, therapeutic potential, and clinical progress

Qian Y, Liu C, Zeng X, Li LC. RNAa: Mechanisms, therapeutic potential, and clinical progress. Molecular Therapy Nucleic Acids. 2025 Feb 21;36(2):102494. DOI: 10.1016/j.omtn.2025.102494

SCAD Delivery Platform: A Novel Approach for Efficient CNS and Extrahepatic Oligonucleotide Therapeutics

Kang M, Lin WH, Xu F, Zhou X, Si C, Liu K, Dai J, He J, Schacht I, Li Y, Li LC. SCAD Delivery Platform: A Novel Approach for Efficient CNS and Extrahepatic Oligonucleotide Therapeutics. bioRxiv. 2024:2024-10. DOI: 10.1101/2024.10.29.620792

Upregulation of LHPP by saRNA inhibited hepatocellular cancer cell proliferation and xenograft tumor growth

Bi CQ, Kang T, Qian YK, Kang MR, Zeng XH, Li LC. Upregulation of LHPP by saRNA inhibited hepatocellular cancer cell proliferation and xenograft tumor growth. PLoS ONE 2024 19(5): e0299522, DOI: 10.1371/journal.pone.0299522

RAG-17: A Novel siRNA Conjugate Demonstrating Efficacy in Late-Stage Treatment of SOD1G93A ALS mice

Duan C, Kang M, Liu K, Gan Z, Li G, Chen J, Schacht I, Place RF, Li LC. RAG-17: A Novel siRNA Conjugate Demonstrating Efficacy in Late-Stage Treatment of SOD1G93A ALS mice. bioRxiv. 2023:2023-11. DOI: 10.1101/2023.11.23.568255

Intrathecal administration of a novel siRNA modality extends survival and improves motor function in the SOD1G93A ALS mouse model

Duan C, Kang M, Pan X, Gan Z, Huang V, Li G, Place RF, Li LC. Intrathecal administration of a novel siRNA modality extends survival and improves motor function in the SOD1G93A ALS mouse model. Molecular Therapy-Nucleic Acids. 2024 Mar 12;35(1). DOI: 10.1016/j.omtn.2024.102147. PMID: 38435120; PMCID: PMC10907209.

Small activating RNA activation of ATOH1 promotes regeneration of human inner ear hair cells.

Zhang YL, Kang M, Wu JC, Xie MY, Xue RY, Tang Q, Yang H, Li LC. Small activating RNA activation of ATOH1 promotes regeneration of human inner ear hair cells. Bioengineered. 2022 Mar 1;13(3):6729-39. DOI: 10.1080/21655979.2022.2045835

Small activating RNA induced expression of VHL gene in renal cell carcinoma.

Kang MR, Park KH, Lee CW, Lee MY, Han SB, Li LC, Kang JS. Small activating RNA induced expression of VHL gene in renal cell carcinoma. The International Journal of Biochemistry & Cell Biology. 2018 Apr 1;97:36-42.

saRNA-guided Ago2 targets the RITA complex to promoters to stimulate transcription.

Portnoy V, Lin SH, Li KH, Burlingame A, Hu ZH, Li H, Li LC. saRNA-guided Ago2 targets the RITA complex to promoters to stimulate transcription. Cell research. 2016 Mar;26(3):320-35.

Inducing gene expression by targeting promoter sequences using small activating RNAs.

Wang J, Place RF, Portnoy V, Huang V, Kang MR, Kosaka M, Ho MK, Li LC. Inducing gene expression by targeting promoter sequences using small activating RNAs. Journal of biological methods. 2015 Mar 11;2(1).

Identification of small activating RNAs that enhance endogenous OCT4 expression in human mesenchymal stem cells.

Wang J, Huang V, Ye L, Bárcena A, Lin G, Lue TF, Li LC. Identification of small activating RNAs that enhance endogenous OCT4 expression in human mesenchymal stem cells. Stem cells and development. 2015 Feb 1;24(3):345-53.

Induction of NANOG expression by targeting promoter sequence with small activating RNA antagonizes retinoic acid-induced differentiation.

Wang X, Wang J, Huang V, Place RF, Li LC. Induction of NANOG expression by targeting promoter sequence with small activating RNA antagonizes retinoic acid-induced differentiation. Biochemical Journal. 2012 May 1;443(3):821-8.

Small activating RNA-guided iNOS up-regulation improves erectile function of diabetic rats

Wang T, Li M, Yuan H, Zhan Y, Xu H, Wang S, Yang W, Liu J, Ye Z, Li LC. saRNA guided iNOS up-regulation improves erectile function of diabetic rats. The Journal of urology. 2013 Aug;190(2):790-8.

Targeted p21 WAF1/CIP1 activation by RNAa inhibits hepatocellular carcinoma cells.

Kosaka M, Kang MR, Yang G and Li LC. Targeted p21 WAF1/CIP1 activation by RNAa inhibits hepatocellular carcinoma cells. Nucleic Acid Ther 2012 2012 Oct; 22(5):335-43.

Intravesical delivery of small activating RNA formulated into lipid nanoparticles inhibits orthotopic bladder tumor growth

Kang MR, Yang G, Place RF, Charisse K, Epstein-Barash H, Manoharan M, Li LC. Intravesical delivery of small activating RNA formulated into lipid nanoparticles inhibits orthotopic bladder tumor growth. Cancer research. 2012 Oct 1;72(19):5069-79.

Targeted induction of endogenous NKX3-1 by small activating RNA inhibits prostate tumor growth.

Ren S, Kang MR, Wang J, Huang V, Place RF, Sun Y, Li LC. Targeted induction of endogenous NKX3‐1 by small activating RNA inhibits prostate tumor growth. The Prostate. 2013 Oct;73(14):1591-601.

Formulation of small activating RNA into lipidoid nanoparticles inhibits xenograft prostate tumor growth by inducing p21 expression

Place RF, Wang J, Noonan EJ, Meyers R, Manoharan M, Charisse K, Duncan R, Huang V, Wang X, Li LC. Formulation of small activating RNA into lipidoid nanoparticles inhibits xenograft prostate tumor growth by inducing p21 expression. Molecular Therapy-Nucleic Acids. 2012 Jan 1;1.

Antitumor effect of dsRNA-induced p21WAF1/CIP1 gene activation in human bladder cancer cells.

Chen Z, Place RF, Jia ZJ, Pookot D, Dahiya R, Li LC. Antitumor effect of dsRNA-induced p21WAF1/CIP1 gene activation in human bladder cancer cells. Molecular cancer therapeutics. 2008 Mar 1;7(3):698-703.

MicroRNA-373 induces expression of genes with complementary promoter sequences.

Place RF, Li LC, Pookot D, Noonan EJ, Dahiya R. MicroRNA-373 induces expression of genes with complementary promoter sequences. Proceedings of the National Academy of Sciences. 2008 Feb 5;105(5):1608-13.

Small double-stranded RNAs induce transcriptional activation in human cells.

Li LC, Okino ST, Zhao H, Pookot D, Place RF, Urakami S, Enokida H, Dahiya R. Small dsRNAs induce transcriptional activation in human cells. Proceedings of the National Academy of Sciences. 2006 Nov 14;103(46):17337-42.

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Presentations

RAG-17, a Novel siRNA Therapy for SOD1-ALS: Safety and Preliminary Efficacy from a First-in-human Trial (Abstract)

Event: American Academy of Neurology (AAN) 2025 Annual Meeting (San Diego) View Abstract View News:…
April 9, 2025
PresentationsRAG-01 Presentation

First-in-human study of RAG-01, a novel small activating RNA therapeutic in BCG failure Non-Muscle Invasive Bladder Cancer (NMIBC) patients(Abstract)

Event:40th Annual Congress of the European Association of Urology (EAU 2025) (Spain) View Abstract View…
March 24, 2025
Presentations

RAG-17: A Promising Novel Gene Silencing Therapy for SOD1-ALS Preliminary Safety and Efficacy Data from a First-in-Human Trial (Abstract)

Event: Society for Neuroscience Annual Meeting 2024 (Neuroscience 2024) (San Diego) View Abstract
October 7, 2024