Journal Article

<i>Ex vivo</i> gene therapy for HIV-1 treatment

Lisa J. Scherer and John J. Rossi

in Human Molecular Genetics

Volume 20, issue R1, pages R100-R107
Published in print April 2011 | ISSN: 0964-6906
Published online April 2011 | e-ISSN: 1460-2083 | DOI: http://dx.doi.org/10.1093/hmg/ddr160
Ex vivo gene therapy for HIV-1 treatment

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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.

Journal Article.  5751 words.  Illustrated.

Subjects: Genetics and Genomics

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