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Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy
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Published on: September 5, 2016

Targeted gene disruption to cure HIV.

Daniel Stone1, Hans-Peter Kiem, Keith R Jerome

  • 1Vaccine and Infectious Disease Division, Fred Hutchinson Cancer Research Center, Department of Medicine, University of Washington, Seattle, Washington 98109, USA.

Current Opinion in HIV and AIDS
|March 13, 2013
PubMed
Summary

Gene disruption strategies show promise for curing HIV. Targeting host and viral genes, as seen in the "Berlin patient," offers a potential one-shot therapy for HIV infection.

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Area of Science:

  • Genetics
  • Virology
  • Immunology

Background:

  • Recent clinical research indicates a potential HIV cure in a patient with lymphoma who received a bone marrow transplant with a disrupted CCR5 allele.
  • This case highlights the possibility of eliminating HIV replication through targeted genetic modifications.

Purpose of the Study:

  • To review and discuss gene disruption approaches for curing HIV infection.
  • To explore strategies for disrupting host and viral genes involved in HIV replication and pathogenesis.

Main Methods:

  • Discussion of precise and imprecise gene disruption techniques.
  • Overview of site-specific recombination strategies.
  • Explanation of engineered nucleases (ZFNs, TALENs, homing endonucleases) and recombinases for targeted DNA modification.

Main Results:

  • The 'Berlin patient' case suggests targeted gene disruption can lead to an HIV cure.
  • Advances in gene disruption technologies are paving the way for novel anti-HIV therapies.

Conclusions:

  • Gene disruption offers a potential curative therapy for HIV, with broad implications for millions of infected individuals.
  • An efficient 'one-shot' curative therapy could provide a drug-free future for patients and reduce healthcare costs associated with long-term antiretroviral therapy.