Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral... Zinc Finger Nucleases (ZFNs) represent powerful tools for genome engineering in human cells. Yet, the full potential of this technology requires solving the challenge of delivering the required machinery to the relevant cell types. Here we exploited the infectivity of integrase-defective lentiviral vectors (IDLV) to express ZFNs and provide the template DNA for gene correction in target cells. IDLV-mediated delivery supported high rates of gene editing in different cell types. IDLVs also mediated site-specific gene addition via a process that required ZFN cleavage and homologous template DNA, thus permitting the design of a platform that can target the insertion of transgenes into a pre-determined genomic site. Site-specific gene addition was observed at previously unattained levels in a panel of human cells, including hematopoietic and embryonic stem cells, allowing the rapid isolation of clonogenic cells with the desired genetic modification. These results open new avenues for experimental biology, biotechnology and medicine.</P> http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png "Blood Cells, Molecules and Diseases" Elsevier

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

Loading next page...
 
/lp/elsevier/gene-editing-in-human-stem-cells-using-zinc-finger-nucleases-and-7o08xd02zQ
Publisher
Elsevier
Copyright
Copyright © 2007 Elsevier Ltd
ISSN
1079-9796
eISSN
1096-0961
DOI
10.1016/j.bcmd.2007.10.064
Publisher site
See Article on Publisher Site

Abstract

Zinc Finger Nucleases (ZFNs) represent powerful tools for genome engineering in human cells. Yet, the full potential of this technology requires solving the challenge of delivering the required machinery to the relevant cell types. Here we exploited the infectivity of integrase-defective lentiviral vectors (IDLV) to express ZFNs and provide the template DNA for gene correction in target cells. IDLV-mediated delivery supported high rates of gene editing in different cell types. IDLVs also mediated site-specific gene addition via a process that required ZFN cleavage and homologous template DNA, thus permitting the design of a platform that can target the insertion of transgenes into a pre-determined genomic site. Site-specific gene addition was observed at previously unattained levels in a panel of human cells, including hematopoietic and embryonic stem cells, allowing the rapid isolation of clonogenic cells with the desired genetic modification. These results open new avenues for experimental biology, biotechnology and medicine.</P>

Journal

"Blood Cells, Molecules and Diseases"Elsevier

Published: Mar 1, 2008

There are no references for this article.

You’re reading a free preview. Subscribe to read the entire article.


DeepDyve is your
personal research library

It’s your single place to instantly
discover and read the research
that matters to you.

Enjoy affordable access to
over 18 million articles from more than
15,000 peer-reviewed journals.

All for just $49/month

Explore the DeepDyve Library

Search

Query the DeepDyve database, plus search all of PubMed and Google Scholar seamlessly

Organize

Save any article or search result from DeepDyve, PubMed, and Google Scholar... all in one place.

Access

Get unlimited, online access to over 18 million full-text articles from more than 15,000 scientific journals.

Your journals are on DeepDyve

Read from thousands of the leading scholarly journals from SpringerNature, Elsevier, Wiley-Blackwell, Oxford University Press and more.

All the latest content is available, no embargo periods.

See the journals in your area

DeepDyve

Freelancer

DeepDyve

Pro

Price

FREE

$49/month
$360/year

Save searches from
Google Scholar,
PubMed

Create folders to
organize your research

Export folders, citations

Read DeepDyve articles

Abstract access only

Unlimited access to over
18 million full-text articles

Print

20 pages / month

PDF Discount

20% off