Literature DB >> 8712765

Gene transfer to hematopoietic stem cells: implications for gene therapy of human disease.

C E Dunbar1.   

Abstract

Transfer of new genetic material to hematopoietic stem cells and expression of the gene product in daughter cells of various lineages is an exciting approach to the treatment of congenital and acquired human diseases. This review summarizes the current status of retroviral and adeno-associated viral vectors for gene transfer to human hematopoietic cells, including extensive preclinical data as well as preliminary results from ongoing clinical trials.

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Year:  1996        PMID： 8712765     DOI： 10.1146/annurev.med.47.1.11

Source DB:  PubMed          Journal:  Annu Rev Med        ISSN： 0066-4219            Impact factor:   13.739


  10 in total

1.  Osteoblast-specific gene expression after transplantation of marrow cells: implications for skeletal gene therapy.

Authors:  Z Hou; Q Nguyen; B Frenkel; S K Nilsson; M Milne; A J van Wijnen; J L Stein; P Quesenberry; J B Lian; G S Stein
Journal:  Proc Natl Acad Sci U S A       Date:  1999-06-22       Impact factor: 11.205

Review 2.  Hematopoietic stem cell engineering at a crossroads.

Authors:  Isabelle Rivière; Cynthia E Dunbar; Michel Sadelain
Journal:  Blood       Date:  2011-11-17       Impact factor: 22.113

3.  Microscale vortex-assisted electroporator for sequential molecular delivery.

Authors:  Dwayne A L Vickers; Soojung Claire Hur
Journal:  J Vis Exp       Date:  2014-08-07       Impact factor: 1.355

4.  Avoidance of stimulation improves engraftment of cultured and retrovirally transduced hematopoietic cells in primates.

Authors:  M Takatoku; S Sellers; B A Agricola; M E Metzger; I Kato; R E Donahue; C E Dunbar
Journal:  J Clin Invest       Date:  2001-08       Impact factor: 14.808

Review 5.  Editing outside the body: Ex vivo gene-modification for β-hemoglobinopathy cellular therapy.

Authors:  Tolulope O Rosanwo; Daniel E Bauer
Journal:  Mol Ther       Date:  2021-10-08       Impact factor: 11.454

Review 6.  Development and clinical translation of ex vivo gene therapy.

Authors:  Xiaomo Wu; Xiaorong He; Fahui Liu; Xiaochang Jiang; Ping Wang; Jinyan Zhang; Ju Jiang
Journal:  Comput Struct Biotechnol J       Date:  2022-06-11       Impact factor: 6.155

7.  Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice.

Authors:  Alex H Chang; Matthias T Stephan; Leszek Lisowski; Michel Sadelain
Journal:  Mol Ther       Date:  2008-08-05       Impact factor: 11.454

Review 8.  Supplying clotting factors from hematopoietic stem cell-derived erythroid and megakaryocytic lineage cells.

Authors:  Michel Sadelain; Alex Chang; Leszek Lisowski
Journal:  Mol Ther       Date:  2009-10-20       Impact factor: 11.454

9.  High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells.

Authors:  H Mochizuki; J P Schwartz; K Tanaka; R O Brady; J Reiser
Journal:  J Virol       Date:  1998-11       Impact factor: 5.103

10.  A novel competitive repopulation strategy to quantitate engraftment of ex vivo manipulated murine marrow cells in submyeloablated hosts.

Authors:  Brandon K Wyss; Justin L Meyers; Anthony L Sinn; Shanbao Cai; Karen E Pollok; W Scott Goebel
Journal:  Exp Hematol       Date:  2008-02-04       Impact factor: 3.084
  10 in total

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