scholarly journals In Vivo Gene Transfer into Adult Stem Cells in Unconditioned Mice by in Situ Delivery of a Lentiviral Vector

2006 ◽  
Vol 14 (4) ◽  
pp. 514-524 ◽  
Author(s):  
D. Nicole Worsham ◽  
Todd Schuesler ◽  
Christof von Kalle ◽  
Dao Pan
Keyword(s):  
Stem Cells ◽  
Gene Transfer ◽  
Adult Stem Cells ◽  
Lentiviral Vector ◽  
In Vivo Gene Transfer

scholarly journals Robust In Vivo Transduction of Nervous System and Neural Stem Cells by Early Gestational Intra Amniotic Gene Transfer Using Lentiviral Vector

2010 ◽  
Vol 18 (9) ◽  
pp. 1615-1623 ◽  
Author(s):  
David H Stitelman ◽  
Masayuki Endo ◽  
Archana Bora ◽  
Nidal Muvarak ◽  
Philip W Zoltick ◽  
...  
Keyword(s):  
Stem Cells ◽  
Nervous System ◽  
Gene Transfer ◽  
Neural Stem Cells ◽  
Lentiviral Vector

Successful treatment of murine β-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells

Blood ◽  
2003 ◽  
Vol 102 (2) ◽  
pp. 506-513 ◽  
Author(s):  
Derek A. Persons ◽  
Esther R. Allay ◽  
Nobukuni Sawai ◽  
Phillip W. Hargrove ◽  
Thomas P. Brent ◽  
...  
Keyword(s):  
Stem Cells ◽  
Gene Transfer ◽  
Hematopoietic Stem Cells ◽  
Bone Marrow Cells ◽  
Lentiviral Vector ◽  
Drug Resistant ◽  
Hematopoietic Stem ◽  
In Vivo Selection ◽  
Selection Of

AbstractSuccessful gene therapy of β-thalassemia will require replacement of the abnormal erythroid compartment with erythropoiesis derived from genetically corrected, autologous hematopoietic stem cells (HSCs). However, currently attainable gene transfer efficiencies into human HSCs are unlikely to yield sufficient numbers of corrected cells for a clinical benefit. Here, using a murine model of β-thalassemia, we demonstrate for the first time that selective enrichment in vivo of transplanted, drug-resistant HSCs can be used therapeutically and may therefore be a useful approach to overcome limiting gene transfer. We used an oncoretroviral vector to transfer a methylguanine methyltransferase (MGMT) drug-resistance gene into normal bone marrow cells. These cells were transplanted into β-thalassemic mice given nonmyeloablative pretransplantation conditioning with temozolomide (TMZ) and O6-benzylguanine (BG). A majority of mice receiving 2 additional courses of TMZ/BG demonstrated in vivo selection of the drug-resistant cells and amelioration of anemia, compared with untreated control animals. These results were extended using a novel γ-globin/MGMT dual gene lentiviral vector. Following drug treatment, normal mice that received transduced cells had an average 67-fold increase in γ-globin expressing red cells. These studies demonstrate that MGMT-based in vivo selection may be useful to increase genetically corrected cells to therapeutic levels in patients with β-thalassemia.

scholarly journals In vivo gene transfer to kidney by lentiviral vector

2002 ◽  
Vol 61 (1) ◽  
pp. S32-S36 ◽  
Author(s):  
G. Luca Gusella ◽  
Elena Fedorova ◽  
Daniele Marras ◽  
Paul E. Klotman ◽  
Mary E. Klotman
Keyword(s):  
Gene Transfer ◽  
Lentiviral Vector ◽  
In Vivo Gene Transfer

scholarly journals In Vivo Gene Transfer Using a Nonprimate Lentiviral Vector Pseudotyped with Ross River Virus Glycoproteins

2002 ◽  
Vol 76 (18) ◽  
pp. 9378-9388 ◽  
Author(s):  
Yubin Kang ◽  
Colleen S. Stein ◽  
Jason A. Heth ◽  
Patrick L. Sinn ◽  
Andrea K. Penisten ◽  
...  
Keyword(s):  
Gene Transfer ◽  
G Protein ◽  
Lentiviral Vector ◽  
Transduction Efficiency ◽  
Ross River Virus ◽  
Immunodeficiency Virus ◽  
Vesicular Stomatitis ◽  
Pseudotyped Vector ◽  
In Vivo Gene Transfer

ABSTRACT Vectors derived from lentiviruses provide a promising gene delivery system. We examined the in vivo gene transfer efficiency and tissue or cell tropism of a feline immunodeficiency virus (FIV)-based lentiviral vector pseudotyped with the glycoproteins from Ross River Virus (RRV). RRV glycoproteins were efficiently incorporated into FIV virions, generating preparations of FIV vector, which after concentration attain titers up to 1.5 × 108 TU/ml. After systemic administration, RRV-pseudotyped FIV vectors (RRV/FIV) predominantly transduced the liver of recipient mice. Transduction efficiency in the liver with the RRV/FIV was ca. 20-fold higher than that achieved with the vesicular stomatitis virus G protein (VSV-G) pseudotype. Moreover, in comparison to VSV-G, the RRV glycoproteins caused less cytotoxicity, as determined from the levels of glutamic pyruvic transaminase and glutamic oxalacetic transaminase in serum. Although hepatocytes were the main liver cell type transduced, nonhepatocytes (mainly Kupffer cells) were also transduced. The percentages of the transduced nonhepatocytes were comparable between RRV and VSV-G pseudotypes and did not correlate with the production of antibody against the transgene product. After injection into brain, RRV/FIV preferentially transduced neuroglial cells (astrocytes and oligodendrocytes). In contrast to the VSV-G protein that targets predominantly neurons, <10% of the brain cells transduced with the RRV pseudotyped vector were neurons. Finally, the gene transfer efficiencies of RRV/FIV after direct application to skeletal muscle or airway were also examined and, although transgene-expressing cells were detected, their proportions were low. Our data support the utility of RRV glycoprotein-pseudotyped FIV lentiviral vectors for hepatocyte- and neuroglia-related disease applications.

In vivo gene transfer to hematopoietic and mesenchymal stromal cells by lentiviral vector

2008 ◽  
Vol 40 (2) ◽  
pp. 278
Author(s):  
Irina Nifontova ◽  
Daria Svinareva ◽  
Natalia Sats ◽  
Vadim Surin ◽  
Nina Drize
Keyword(s):  
Gene Transfer ◽  
Mesenchymal Stromal Cells ◽  
Stromal Cells ◽  
Lentiviral Vector ◽  
In Vivo Gene Transfer

Lentiviral vector–mediated, in vivo gene transfer to the tracheobronchial tree in fetal rabbits

2005 ◽  
Vol 40 (12) ◽  
pp. 1817-1821 ◽  
Author(s):  
Erik D. Skarsgard ◽  
Louis Huang ◽  
Stacy C. Reebye ◽  
Adam Y. Yeung ◽  
William W. Jia
Keyword(s):  
Gene Transfer ◽  
Lentiviral Vector ◽  
Tracheobronchial Tree ◽  
In Vivo Gene Transfer
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