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PUBLICATIONS

Publication - Abstract

May 13, 2021
Nature Communications

Tissue-specific Activation of Gene Expression by the Synergistic Activation Mediator (SAM) CRISPRa System in Mice

C. Hunt, S.A. Hartford, G. Gong, et al

mRNA Genetic Medicine Other Applications

The paper demonstrates PNI's mRNA-LNP as highly capable of inducing strong levels of upregulation in target genes with activation levels six to nine fold dependent on amount of gRNA packaged. The paper validates the SAM system in mice as a suitable in vivo model encompassing both...
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Publication - Abstract

Nov 18, 2020
Science Advances

CRISPR- Cas9 Genome Editing Using Targeted Lipid Nanoparticles for Cancer Therapy

D. Rosenblum, A. Gutkin, R. Kedmi, S. Ramishetti, N. Veiga, A.M. Jacobi and D. Peer et. al.

mRNA Nucleic Acid Lipid Nanoparticles Oncology

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Publication - Abstract

May 22, 2020
SLAS Discovery

Arrayed CRISPR Screening Identifies Novel Targets That Enhance the Productive Delivery of mRNA by MC3-Based Lipid Nanoparticles

D. Ross-Thriepland, A. Bornot, L. Butler, A. Desai, H. Jaiswal, S. Peel, M.R. Hunter, U. Odunze, B. Isherwood and D. Gianni

Modified messenger RNAs (mRNAs) hold great potential as therapeutics by using the body’s own processes for protein production. However, a key challenge is efficient delivery of therapeutic mRNA to the cell cytosol and productive protein translation. Lipid nanoparticles (LNP...
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Publication - Summary

Feb 27, 2018
Cell Reports

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

J. Finn, A. Smith, M. Patel, L. Shaw, M. Youniss, J. Heteren, T. Dirstine, C. Ciullo, R. Lescarbeau, J. Seitzer, R. Shah, A. Shah, D. Ling, J. Growe, M. Pink, E. Rohde, K. Wood, W. Salomon, W. Harrington, C. Dombrowski, W. Strapps, Y. Chang, D. Morrissey

Other Payloads mRNA Nucleic Acid Lipid Nanoparticles

Hereditary transthryretin amyloidosis is a rare disease caused by mutations in the gene encoding the protein transthyretin (TTR), causing it to misfold into amyloid plaques, leading to debilitating symptoms. In 2018,...
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Publication - Summary

Dec 16, 2016
Angewandte Chemie International Edition

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

Miller JB, Zhang S, Kos P, Xiong H, Zhou K, Perelman SS, Zhu H, Siegwart DJ

Other Payloads mRNA Nucleic Acid Lipid Nanoparticles

The CRISPR/Cas (clustered regularly interspaced short palindromic repeat/CRISPR-associated protein) gene-editing tool allows for sequence-specific cuts to genomic DNA, which can result in permanent silencing of a gene, or replacement of gene sequen...
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Publication - Abstract

Feb 11, 2021
Nano Letters

Natural or Synthetic RNA Delivery: A Stoichiometric Comparison of Extracellular Vesicles and Synthetic Nanoparticles

D.E. Murphy, O.G. de Jong, M.J.W. Evers, M. Nurazizah, Y.M. Schiffelers and P. Vader

mRNA Nucleic Acid Lipid Nanoparticles Other Applications

RNA therapeutics have high potential that is yet to be fully realized, largely due to challenges involved in the appropriate delivery to target cells. Extracellular vesicles (EVs) are lipid bound nanoparticles released by cells of all types and possess numerous features that may ...
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Publication - Abstract

Jul 07, 2017
ACS Applied Materials & Interfaces

Biodegradable Amino-ester Nanomaterials for Cas9 mRNA Delivery in Vitro and in Vivo

X. Zhang, B. Li, X. Luo, W. Zhao, J. Jiang, C. Zhang, M. Gao, X. Chen and Y. Dong

mRNA Nucleic Acid Lipid Nanoparticles Genetic Medicine

Efficient and safe delivery of the CRISPR/Cas system is one of the key challenges for genome-editing applications in humans. Herein, we designed and synthesized a series of biodegradable lipidlike compounds containing ester groups for the delivery ...
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POSTERS

Poster

Jun 14, 2023

Lipid Nanoparticle-Mediated Gene Editing of Human Primary T Cells and Off-Target Analysis of the CRISPR-Cas9 Indels

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Poster

Dec 04, 2017

Lipid Nanoparticles Deliver Nucleic Acids, Including CRISPR Components, in Primary Neurons

mRNA DNA Nucleic Acid Lipid Nanoparticles

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VIDEOS & WEBINARS

Video

Jul 25, 2016

Improved CRISPR Genome Editing using crRNA:tracrRNA complexes and Cas9 | Ashley Jacobi, IDT

Other Payloads DNA mRNA

Improved CRISPR Genome Editing using crRNA:tracrRNA complexes and Cas9 | Ashley Jacobi, IDT

Resource Center Search

PUBLICATIONS

Tissue-specific Activation of Gene Expression by the Synergistic Activation Mediator (SAM) CRISPRa System in Mice

CRISPR- Cas9 Genome Editing Using Targeted Lipid Nanoparticles for Cancer Therapy

Arrayed CRISPR Screening Identifies Novel Targets That Enhance the Productive Delivery of mRNA by MC3-Based Lipid Nanoparticles

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

Natural or Synthetic RNA Delivery: A Stoichiometric Comparison of Extracellular Vesicles and Synthetic Nanoparticles

Biodegradable Amino-ester Nanomaterials for Cas9 mRNA Delivery in Vitro and in Vivo

POSTERS

Lipid Nanoparticle-Mediated Gene Editing of Human Primary T Cells and Off-Target Analysis of the CRISPR-Cas9 Indels

Lipid Nanoparticles Deliver Nucleic Acids, Including CRISPR Components, in Primary Neurons

VIDEOS & WEBINARS

Improved CRISPR Genome Editing using crRNA:tracrRNA complexes and Cas9 | Ashley Jacobi, IDT

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