title: Progress toward curing HIV infection with hematopoietic cell transplantation.
abstract: HIV-1 infection afflicts more than 35 million people worldwide, according to 2014 estimates from the World Health Organization. For those individuals who have access to antiretroviral therapy, these drugs can effectively suppress, but not cure, HIV-1 infection. Indeed, the only documented case for an HIV/AIDS cure was a patient with HIV-1 and acute myeloid leukemia who received allogeneic hematopoietic cell transplantation (HCT) from a graft that carried the HIV-resistant CCR5-∆32/∆32 mutation. Other attempts to establish a cure for HIV/AIDS using HCT in patients with HIV-1 and malignancy have yielded mixed results, as encouraging evidence for virus eradication in a few cases has been offset by poor clinical outcomes due to the underlying cancer or other complications. Such clinical strategies have relied on HIV-resistant hematopoietic stem and progenitor cells that harbor the natural CCR5-∆32/∆32 mutation or that have been genetically modified for HIV-resistance. Nevertheless, HCT with HIV-resistant cord blood remains a promising option, particularly with inventories of CCR5-∆32/∆32 units or with genetically modified, human leukocyte antigen-matched cord blood.
authors.affiliation: StemCyte international Cord Blood Center, Baldwin Park, CA, USA.
authors.forename: Lawrence D
authors.initials: LD
authors.lastname: Petz
authors.affiliation: Department of Molecular and Cellular Biology, Irell and Manella Graduate School of Biological Sciences, City of Hope, Duarte, CA, USA.
authors.forename: John C
authors.initials: JC
authors.lastname: Burnett
authors.affiliation: Department of Molecular and Cellular Biology, Beckman Research institute, City of Hope, Duarte, CA, USA.
authors.forename: Haitang
authors.initials: H
authors.lastname: Li
authors.affiliation: Department of Molecular and Cellular Biology, Beckman Research institute, City of Hope, Duarte, CA, USA.
authors.forename: Shirley
authors.initials: S
authors.lastname: Li
authors.affiliation: StemCyte international Cord Blood Center, Baldwin Park, CA, USA.
authors.forename: Richard
authors.initials: R
authors.lastname: Tonai
authors.affiliation: CCR5-Δ32/Δ32 Research Department, StemCyte international Cord Blood Center, Baldwin Park, CA, USA.
authors.forename: Milena
authors.initials: M
authors.lastname: Bakalinskaya
authors.affiliation: Department of Stem Cell Transplantation, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Elizabeth J
authors.initials: EJ
authors.lastname: Shpall
authors.affiliation: MD Anderson Cord Blood Bank, Department of Stem Cell Transplantation, University of Texas MD Anderson Cancer Center, Houston, TX, USA.
authors.forename: Sue
authors.initials: S
authors.lastname: Armitage
authors.affiliation: Carolinas Cord Blood Bank, Duke University Medical Center, Durham, NC, USA.
authors.forename: Joanne
authors.initials: J
authors.lastname: Kurtzberg
authors.affiliation: St Louis Cord Blood Bank, SSM Cardinal Glennon Children's Medical Center, St Louis, MO, USA.
authors.forename: Donna M
authors.initials: DM
authors.lastname: Regan
authors.affiliation: Enhance Quality Consulting Inc., Oviedo, FL, USA.
authors.forename: Pamela
authors.initials: P
authors.lastname: Clark
authors.affiliation: Cell Therapy Service and Cord Blood Bank, Banc de Sang i Teixits, Barcelona, Spain.
authors.forename: Sergio
authors.initials: S
authors.lastname: Querol
authors.affiliation: BMT/Hematologic Malignancies, University of Colorado, Aurora, CO, USA.
authors.forename: Jonathan A
authors.initials: JA
authors.lastname: Gutman
authors.affiliation: Immunobiology and Observational Research, CIBMTR, Minneapolis, MN, USA.
authors.forename: Stephen R
authors.initials: SR
authors.lastname: Spellman
authors.affiliation: National Marrow Donor Program/Be The Match, Minneapolis, MN, USA.
authors.forename: Loren
authors.initials: L
authors.lastname: Gragert
authors.affiliation: Department of Molecular and Cellular Biology, Irell and Manella Graduate School of Biological Sciences, City of Hope, Duarte, CA, USA.
authors.forename: John J
authors.initials: JJ
authors.lastname: Rossi
keywords: CCR5 mutation; CCR5-∆32/∆32 cord blood inventory; curing HIV infection; genetic modification of stem cells; hematopoietic cell transplantation