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Non-Viral Gold Nanoparticle Mediated...

Cunningham, Rachel,

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Non-Viral Gold Nanoparticle Mediated Delivery of CRISPR-Cas9 Ribonucleoprotein and Long DNA Transgenes into Primary Blood Cells /

Record Type:	Electronic resources : Monograph/item
Title/Author:	Non-Viral Gold Nanoparticle Mediated Delivery of CRISPR-Cas9 Ribonucleoprotein and Long DNA Transgenes into Primary Blood Cells // Rachel Cunningham.
Author:	Cunningham, Rachel,
Description:	1 electronic resource (87 pages)
Notes:	Source: Dissertations Abstracts International, Volume: 87-01, Section: B.
Contained By:	Dissertations Abstracts International87-01B.
Subject:	Nanotechnology. -
Online resource:	https://pqdd.sinica.edu.tw/twdaoapp/servlet/advanced?query=32116243
ISBN:	9798288833427

Non-Viral Gold Nanoparticle Mediated Delivery of CRISPR-Cas9 Ribonucleoprotein and Long DNA Transgenes into Primary Blood Cells /
Cunningham, Rachel,

Non-Viral Gold Nanoparticle Mediated Delivery of CRISPR-Cas9 Ribonucleoprotein and Long DNA Transgenes into Primary Blood Cells /Rachel Cunningham. - 1 electronic resource (87 pages)

Source: Dissertations Abstracts International, Volume: 87-01, Section: B.

Gene editing using CRISPR systems has gained traction for its potential to treat various diseases. However, current gene editing therapies suffer from lack of affordable, scalable and synthetic delivery, especially for complex cargo such as the CRISPR ribonucleoprotein (RNP) complex and DNA templates required for insertion of specific genomic sequences. Development of an effective non-viral approach to RNP delivery could overcome these limitations and would be transformative for clinical translation and research. We previously reported a simple, synthetic gold-based nanoparticle which can deliver various CRISPR systems as RNP into primary hematopoietic stem and progenitor cells without the need for complex protein engineering (CRISPR-AuNP). Here, we describe a gold-based nanoparticle to simultaneously deliver CRISPR systems and large DNA templates which can encode transgenes (HDT-CRISPR-AuNP). HDT-CRISPR-AuNP can carry templates as long as ~2.1kb, with potential for larger cargo. We evaluated HDT-CRISPR-AuNP for gene editing at two loci of interest with different transgenes. These nanoparticles were able to successfully engineer primary human T cells and hematopoietic stem and progenitor cells with insertion of an antigen-specific T-cell receptor (TCR) transgene. This proof-of-concept immune engineering study shows transgene integration and expression from various hematopoietic lineages and suggests a potential for efficacy in vivo.

English

ISBN: 9798288833427Subjects--Topical Terms:

526235
Nanotechnology.
Subjects--Index Terms:

Drug delivery

Non-Viral Gold Nanoparticle Mediated Delivery of CRISPR-Cas9 Ribonucleoprotein and Long DNA Transgenes into Primary Blood Cells /
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520 $a Gene editing using CRISPR systems has gained traction for its potential to treat various diseases. However, current gene editing therapies suffer from lack of affordable, scalable and synthetic delivery, especially for complex cargo such as the CRISPR ribonucleoprotein (RNP) complex and DNA templates required for insertion of specific genomic sequences. Development of an effective non-viral approach to RNP delivery could overcome these limitations and would be transformative for clinical translation and research. We previously reported a simple, synthetic gold-based nanoparticle which can deliver various CRISPR systems as RNP into primary hematopoietic stem and progenitor cells without the need for complex protein engineering (CRISPR-AuNP). Here, we describe a gold-based nanoparticle to simultaneously deliver CRISPR systems and large DNA templates which can encode transgenes (HDT-CRISPR-AuNP). HDT-CRISPR-AuNP can carry templates as long as ~2.1kb, with potential for larger cargo. We evaluated HDT-CRISPR-AuNP for gene editing at two loci of interest with different transgenes. These nanoparticles were able to successfully engineer primary human T cells and hematopoietic stem and progenitor cells with insertion of an antigen-specific T-cell receptor (TCR) transgene. This proof-of-concept immune engineering study shows transgene integration and expression from various hematopoietic lineages and suggests a potential for efficacy in vivo.
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