Open AccessEx vivo gene therapy for HIV-1 treatment
Author(s) -
Lisa Scherer,
John J. Rossi
Publication year - 2011
Publication title -
human molecular genetics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 2.811
H-Index - 276
eISSN - 1460-2083
pISSN - 0964-6906
DOI - 10.1093/hmg/ddr160
Subject(s) - genetic enhancement , biology , ex vivo , stem cell , cell therapy , immunology , lymphoma , vector (molecular biology) , lentivirus , human immunodeficiency virus (hiv) , virology , in vivo , gene , viral disease , genetics , recombinant dna
Until recently, progress in ex vivo gene therapy (GT) for human immunodeficiency virus-1 (HIV-1) treatment has been incremental. Long-term HIV-1 remission in a patient who received a heterologous stem cell transplant for acquired immunodeficiency syndrome-related lymphoma from a CCR5(-/-) donor, even after discontinuation of conventional therapy, has energized the field. We review the status of current approaches as well as future directions in the areas of therapeutic targets, combinatorial strategies, vector design, introduction of therapeutics into stem cells and enrichment/expansion of gene-modified cells. Finally, we discuss recent advances towards clinical application of HIV-1 GT.
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