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CRISPR/Cas9 Genome Editing01:28

CRISPR/Cas9 Genome Editing

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...
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siRNA - Small Interfering RNAs02:30

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Small interfering RNAs, or siRNAs, are short regulatory RNA molecules that can silence genes post-transcriptionally, as well as the transcriptional level in some cases. siRNAs are important for protecting cells against viral infections and silencing transposable genetic elements.
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CRISPR01:59

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing its transcription or activating the RNA degradation process. The mechanism was discovered by Andrew Fire and Craig Mello in 1998 in plants. Today, it is observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals. This precise cellular mechanism of gene silencing has been developed into a technique that provides an efficient way to identify and determine the...
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RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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Related Experiment Video

Updated: Jul 28, 2025

CIRCLE-Seq for Interrogation of Off-Target Gene Editing
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New Approaches for Targeting PCSK9: Small-Interfering Ribonucleic Acid and Genome Editing.

Reindert F Oostveen1, Amit V Khera2, Sekar Kathiresan2

  • 1Department of Vascular Medicine, Amsterdam Cardiovascular Sciences, Amsterdam UMC, University of Amsterdam, The Netherlands (R.F.O., E.S.G.S., J.J.P.K.).

Arteriosclerosis, Thrombosis, and Vascular Biology
|June 1, 2023
PubMed
Summary

Novel therapies targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) using RNA interference and genome editing offer promising long-acting solutions for patients with very high cardiovascular risk who struggle to meet lipid goals.

Keywords:
apolipoproteincardiologycoronary artery diseasemutationsubtilisin

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Area of Science:

  • Cardiovascular Medicine
  • Genetics
  • Pharmacology

Background:

  • Low-density-lipoprotein cholesterol (LDL-C) reduction is critical for patients at very high cardiovascular risk.
  • Current lipid-lowering therapies often fail to achieve guideline-recommended LDL-C goals.
  • Adherence and chronic care challenges limit the effectiveness of existing treatments.

Purpose of the Study:

  • To review the evolution of targeting PCSK9 (proprotein convertase subtilisin/kexin type 9) for lipid management.
  • To explore novel therapeutic strategies including mRNA, small-interfering RNA, and genome editing.
  • To highlight the potential of these advanced approaches to improve patient outcomes.

Main Methods:

  • Review of historical and current research on PCSK9 inhibition.
  • Discussion of mRNA and small-interfering RNA (siRNA) based therapies.
  • Exploration of genome editing technologies, including CRISPR-base editing.

Main Results:

  • PCSK9 inhibition represents a significant advancement in cardiovascular risk reduction.
  • RNA-based therapies offer sustained and effective LDL-C lowering.
  • Genome editing, exemplified by VERVE-101, shows potential for durable PCSK9 targeting.

Conclusions:

  • Targeting PCSK9 with novel modalities like siRNA and genome editing addresses unmet needs in cardiovascular risk management.
  • These approaches may overcome limitations of current therapies, improving adherence and goal attainment.
  • Future research and clinical trials are essential to realize the full potential of these innovative treatments.