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

2008 ◽  
Vol 40 (2) ◽  
pp. 278 ◽  
Author(s):  
A. Lombardo ◽  
P. Genovese ◽  
C.M. Beausejour ◽  
S. Colleoni ◽  
Y.-L. Lee ◽  
...  
Keyword(s):  
Stem Cells ◽  
Zinc Finger ◽  
Gene Editing ◽  
Lentiviral Vector ◽  
Zinc Finger Nucleases ◽  
Human Stem Cells ◽  
Vector Delivery

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

2007 ◽  
Vol 25 (11) ◽  
pp. 1298-1306 ◽  
Author(s):  
Angelo Lombardo ◽  
Pietro Genovese ◽  
Christian M Beausejour ◽  
Silvia Colleoni ◽  
Ya-Li Lee ◽  
...  
Keyword(s):  
Stem Cells ◽  
Zinc Finger ◽  
Gene Editing ◽  
Lentiviral Vector ◽  
Zinc Finger Nucleases ◽  
Human Stem Cells ◽  
Vector Delivery

scholarly journals Gene Editing of Human Embryonic Stem Cells via an Engineered Baculoviral Vector Carrying Zinc-finger Nucleases

2011 ◽  
Vol 19 (5) ◽  
pp. 942-950 ◽  
Author(s):  
Yuning Lei ◽  
Chi-Lin Lee ◽  
Kye-Il Joo ◽  
Jonathan Zarzar ◽  
Yarong Liu ◽  
...  
Keyword(s):  
Stem Cells ◽  
Embryonic Stem Cells ◽  
Zinc Finger ◽  
Human Embryonic Stem Cells ◽  
Gene Editing ◽  
Embryonic Stem ◽  
Zinc Finger Nucleases

Codon Swapping of Zinc Finger Nucleases Confers Expression in Primary Cells and In Vivo from a Single Lentiviral Vector

2014 ◽  
Vol 14 (5) ◽  
pp. 365-376 ◽  
Author(s):  
Abarrategui-Pontes Cecilia ◽  
Creneguy Alison ◽  
Thinard Reynald ◽  
Fine J. ◽  
Thepenier Virginie ◽  
...  
Keyword(s):  
Zinc Finger ◽  
Lentiviral Vector ◽  
Zinc Finger Nucleases ◽  
Primary Cells

scholarly journals Selection-Independent Generation of Gene Knockout Mouse Embryonic Stem Cells Using Zinc-Finger Nucleases

PLoS ONE ◽  
2011 ◽  
Vol 6 (12) ◽  
pp. e28911 ◽  
Author(s):  
Anna Osiak ◽  
Frank Radecke ◽  
Eva Guhl ◽  
Sarah Radecke ◽  
Nadine Dannemann ◽  
...  
Keyword(s):  
Stem Cells ◽  
Embryonic Stem Cells ◽  
Zinc Finger ◽  
Knockout Mouse ◽  
Gene Knockout ◽  
Embryonic Stem ◽  
Mouse Embryonic Stem Cells ◽  
Zinc Finger Nucleases ◽  
Gene Knockout Mouse

Taking it to the Clinic: Genome Editing for Blood Disorders

Blood ◽  
2014 ◽  
Vol 124 (21) ◽  
pp. SCI-12-SCI-12
Author(s):  
Fyodor D. Urnov
Keyword(s):  
Stem Cells ◽  
Zinc Finger Nucleases ◽  
Proof Of Concept ◽  
Human Stem Cells ◽  
Blood Disorders ◽  
Therapeutic Application ◽  
Hematopoietic Stem ◽  
Monogenic Diseases ◽  
Potential Therapeutic Application ◽  
Cell Genome

Abstract The ability to engineer precise genetic modification of human stem cells offers the promise to extend the range of their potential therapeutic application. This possibility is now being realized via the use of zinc finger nucleases (ZFNs). ZFNs are customizable, sequence-specific endonucleases that can be designed to introduce a discrete cleavage event at any user-chosen location within the stem cell genome. This presentation will describe our progress in translating ZFN-modified hematopoietic stem/progenitor cells (HSPCs) from concept to clinic, focusing on preclinical proof-of-concept studies supporting the development of autologous, genome-edited CD34+ HSPCs as potential treatments for both HIV/AIDS as well as certain monogenic diseases of the blood, such as β-thalassemia. Disclosures Urnov: Sangamo BioSciences: Employment.

Sign in / Sign up

Export Citation Format

Share Document