Related Experiment Video

Updated: Nov 22, 2025

Oral Combinational Antiretroviral Treatment in HIV-1 Infected Humanized Mice
06:07

Oral Combinational Antiretroviral Treatment in HIV-1 Infected Humanized Mice

Published on: October 6, 2022

2.7K

Combinatorial Strategies for Long-term Control of HIV Infection

Daekee Kwon1, Mi-Jung Han1, Kwang-Won Seo1

  • 1Stem Cells and Regenerative Bioengineering Institute in Kangstem Biotech, Gwangmyeong SK TechnoPark, Gyeonggi-do, Seoul, South Korea.

AIDS Reviews
|January 5, 2021
PubMed

Insights

Patient-specific CCR5 gene-edited hematopoietic stem cells (HSC) offer a promising approach for long-term HIV control. Combining gene editing with cellular reprogramming overcomes challenges in matching HSC for transplantation.

Area of Science:

  • * Hematology
  • * Immunology
  • * Gene Therapy

Background:

  • * Acquired Immunodeficiency Syndrome (AIDS) results from chronic Human Immunodeficiency Virus (HIV) infection.
  • * Bone marrow transplantation of hematopoietic stem cells (HSC) with innate C-C chemokine receptor type 5 (CCR5) mutations shows potential for long-term HIV control.
  • * A significant hurdle is the difficulty in obtaining CCR5-mutant HSC that are human leukocyte antigen (HLA)-matched between donor and recipient.

Purpose of the Study:

  • * To review and discuss methods for generating patient-specific CCR5 gene-edited HSC.
  • * To explore the integration of gene editing and cellular reprogramming technologies for HIV therapeutic applications.

Main Methods:

  • * Utilizes advanced gene editing tools: zinc-finger nucleases (ZFN), transcription activator-like effector nucleases (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR).
  • * Integrates cellular reprogramming technologies: somatic cell nuclear transfer (SCNT), induced pluripotent stem cells (iPSC), and direct phenotypic conversion.
  • * Focuses on generating patient-specific CCR5-edited HSC to overcome HLA matching limitations.

Main Results:

  • * The combination of gene editing and cellular reprogramming enables the creation of patient-specific CCR5-edited HSC.
  • * These engineered HSC hold potential for addressing the HLA matching challenge in transplantation.

Conclusions:

  • * Patient-specific CCR5 gene-edited HSC represent a viable strategy for future HIV therapies.
  • * These cellular agents could provide a pathway for long-term control of HIV infection in patients.

More Related Videos

Rapid Screening of HIV Reverse Transcriptase and Integrase Inhibitors05:46

Rapid Screening of HIV Reverse Transcriptase and Integrase Inhibitors

Published on: April 9, 2014

18.1K
Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy12:03

Evaluation of the Efficacy And Toxicity of RNAs Targeting HIV-1 Production for Use in Gene or Drug Therapy

Published on: September 5, 2016

8.1K

Related Concept Videos

Retrovirus Life Cycles01:10

Retrovirus Life Cycles

Retroviruses have a single-stranded RNA genome that undergoes a special form of replication. Once the retrovirus has entered the host cell, an enzyme called reverse transcriptase synthesizes double-stranded DNA from the retroviral RNA genome. This DNA copy of the genome is then integrated into the host’s genome inside the nucleus via an enzyme called integrase. Consequently, the retroviral genome is transcribed into RNA whenever the host’s genome is transcribed, allowing the...
48.4K
Size and Structure of Viral Genomes01:26

Size and Structure of Viral Genomes

Viral genomes exhibit remarkable diversity in size, structure, and composition, influencing their replication strategies and interactions with host cells. These genomes consist of either DNA or RNA and may be linear or circular. Additionally, they can be single-stranded or double-stranded, with each configuration affecting how the virus propagates within a host. RNA viruses, for instance, generally have smaller genomes than DNA viruses, a factor that contributes to their high mutation rates and...
424
Viruses with RNA Genomes01:29

Viruses with RNA Genomes

RNA viruses are categorized into positive-strand, negative-strand, or double-stranded groups based on their genomic structure and replication mechanisms. This classification dictates how they exploit host cellular machinery for protein synthesis and replication. Some RNA viruses also utilize reverse transcription as part of their life cycle, further diversifying their replication strategies.Positive-Strand RNA VirusesPositive-strand RNA viruses have genomes that function directly as messenger...
422