Category: Publications

Publication Spotlight: Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

Posted on 05/28/16 by defeatHIV

Genome editing in hematopoietic stem and progenitor cells (HSPCs) is a promising novel technology for the treatment of many human diseases. Here, we evaluated whether the disruption of the C-C chemokine receptor 5 (CCR5) locus in pigtailed macaque HSPCs by zinc finger nucleases (ZFNs) was feasible. We show that macaque-specific CCR5 ZFNs efficiently induce CCR5 […]

Read more

Publication Spotlight: A curative regimen would decrease HIV prevalence but not HIV incidence unless targeted to an ART-naïve population

Posted on 03/01/16 by defeatHIV

HIV curative strategies currently under development aim to eradicate latent provirus, or prevent viral replication, progression to AIDS, and transmission. The impact of implementing curative programs on HIV epidemics has not been considered. We developed a mathematical model of heterosexual HIV transmission to evaluate the independent and synergistic impact of ART, HIV prevention interventions and […]

Read more

Publication Spotlight: Detection of treatment-resistant infectious HIV after genome-directed antiviral endonuclease therapy

Posted on 02/15/16 by defeatHIV

Incurable chronic viral infections are a major cause of morbidity and mortality worldwide. One potential approach to cure persistent viral infections is via the use of targeted endonucleases. Nevertheless, a potential concern for endonuclease-based antiviral therapies is the emergence of treatment resistance. Here we detect for the first time an endonuclease-resistant infectious virus that is […]

Read more

Publication Spotlight: Safety and Efficacy of Combination Antiretroviral Therapy in Human Immunodeficiency Virus-Infected Adults Undergoing Autologous or Allogeneic Hematopoietic Cell Transplantation for Hematologic Malignancies

Posted on 01/19/16 by defeatHIV

The ability to continue combination antiretroviral therapy (cART) in human immunodeficiency virus (HIV)-infected patients undergoing hematopoietic cell transplantation (HCT) for treatment of hematologic malignancies is likely a critical factor in preventing the establishment of an HIV reservoir in transplanted stem cells. Thus, we studied the feasibility of continued antiretroviral therapy in our HIV-infected patients undergoing […]

Read more

Publication Spotlight: Genetically modified hematopoietic stem cell transplantation for HIV-1-infected patients: can we achieve a cure?

Posted on 02/21/14 by defeatHIV

The cure of a human immunodeficiency virus (HIV)-1-infected patient following allogeneic transplantation from a CCR5-null donor and potential cure of two patients transplanted with CCR5 wild-type hematopoietic stem cells (HSC) have provided renewed optimism that a potential alternative to conventional antiretroviral therapy (ART) is forthcoming. While allogeneic grafts have thus far suggested complete eradication of […]

Read more

Publication Spotlight: Combinatorial anti-HIV gene therapy: using a multipronged approach to reach beyond HAART

Posted on 07/27/13 by defeatHIV

The ‘Berlin Patient’, who maintains suppressed levels of HIV viremia in the absence of antiretroviral therapy, continues to be a standard bearer in HIV eradication research. However, the unique circumstances surrounding his functional cure are not applicable to most HIV(+) patients. To achieve a functional or sterilizing cure in a greater number of infected individuals […]

Read more

Publication Spotlight: Extended Survival of Glioblastoma Patients After Chemoprotective HSC Gene Therapy

Posted on 07/13/12 by defeatHIV
Glioblastoma Patients MRI 1

Extended Survival of Glioblastoma Patients After Chemoprotective HSC Gene Therapy Science Translational Medicine May 2012 Jennifer E. Adair, Brian C. Beard, Grant D. Trobridge, Tobias Neff, Jason K. Rockhill, Daniel L. Silbergeld, Maciej M. Mrugala, and Hans-Peter Kiem MRI of the brain (subject one). Axial T1 post gadolinium sequences at the level of midbrain. A. […]

Read more

Publication Spotlight: Hematopoietic-Stem-Cell-Based Gene Therapy for HIV Disease

Posted on 03/14/12 by defeatHIV

Hematopoietic-Stem-Cell-Based Gene Therapy for HIV Disease Cell Stem Cell Feb 2012 Hans-Peter Kiem, Keith R. Jerome, Steven G. Deeks, Joseph M. McCune Investigators from defeatHIV and the Delaney AIDS Research Enterprise (DARE), two of the three NIAID Martin Delaney Collaboratories, partner to review HSC-based gene therapy strategies for HIV disease – demonstrating how scientists in […]

Read more