Target intelligence / Profile preview

Target mRNAs specified by the 7 shRNA sequences

Molecular classification
Other
01

Overview

Target mRNAs specified by the 7 shRNA sequences refers to a collective set of messenger RNAs (mRNAs) targeted by a multiplexed RNA interference (RNAi) therapeutic strategy, most notably developed for the treatment of HIV-1 infection. This specific approach, pioneered by researchers at the City of Hope and Benitec Biopharma, utilizes a single genetic construct (often a lentiviral vector) to express seven distinct short hairpin RNAs (shRNAs) or microRNA-adapted shRNAs (shRNA-miRs). These seven sequences are designed to simultaneously silence the human CCR5 co-receptor (a key host factor for viral entry) and six essential regions within the HIV-1 viral genome, including the Gag, Pol, Env, Tat, and Vif genes. By targeting multiple sites across both host and viral transcripts, this combinatorial RNAi (co-RNAi) strategy aims to achieve potent viral suppression while significantly increasing the genetic barrier to viral escape, a major challenge for single-target RNAi therapies. Although primarily an investigational tool and a proof-of-concept for multiplexed gene therapy, this target set represents a sophisticated attempt to overcome the high mutation rate of HIV-1 through multi-pronged molecular intervention.

Other names
HIV-1 and CCR5 mRNAs7-shRNA-miR targetsMultiplexed anti-HIV shRNA targetsCombinatorial RNAi targets
02

Mechanism of action

RNA interference (RNAi) mediated degradation of target mRNAs

03

Biological functions

Viral replicationViral entryViral assemblyViral transcriptionHost-pathogen interaction
04

Disease associations

InfectionOther
05

Safety considerations

Off-target effects (silencing of non-target host genes)Saturation of the endogenous RNAi machinery (e.g., Exportin-5, RISC)Immune activation (TLR7/8)Viral escape (minimized by multiplexing but still a theoretical risk)
06

Interacting drugs

Multiplexed 7-shRNA-miR construct

2 more in the full profile.

07

Biomarkers

HIV-1 viral load (p24, RNA)CD4+ T-cell countCCR5 expression levels

Beyond the preview

Go deeper on Target mRNAs specified by the 7 shRNA sequences.

Explore the evidence, development activity, and competitive landscape with Gosset’s full data platform.

Drug pipeline

Full profile access

Explore the programs pursuing this target and their development progress.

  • Drug candidates
  • Developers
  • Development stage

Clinical trials

Full profile access

Follow the clinical studies evaluating therapies directed at this target.

  • Trial design
  • Status
  • Readouts

Competitive landscape

Full profile access

Compare approaches across drug candidates, modalities, and indications.

  • Programs
  • Modalities
  • Indications

Literature & evidence

Full profile access

Investigate the research and source evidence behind target biology and development.

  • Publications
  • Sources
  • Analysis

Patents

Full profile access

Explore patent activity around therapies and technologies addressing this target.

  • Patents
  • Assignees
  • Technologies

Research & analysis

Full profile access

Connect target biology, drug development, and emerging evidence in your research.

  • Biology
  • Development news
  • Analysis

Bring the full picture into focus.

See how Gosset can support your research on Target mRNAs specified by the 7 shRNA sequences.

Explore the full profile

Gosset Free

Get started with Gosset.

Enter your work email and we’ll be in touch with next steps.

Work email preferred.

Book a call