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Related Concept Videos

CRISPR01:59

CRISPR

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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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Epigenetic Regulation01:37

Epigenetic Regulation

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Epigenetic changes alter the physical structure of the DNA without changing the genetic sequence and often regulate whether genes are turned on or off. This regulation ensures that each cell produces only proteins necessary for its function. For example, proteins that promote bone growth are not produced in muscle cells. Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.
X-chromosome...
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What is Genetic Engineering?00:49

What is Genetic Engineering?

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Overview
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RNA Editing02:23

RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Methods of Nuclear Reprogramming01:24

Methods of Nuclear Reprogramming

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Nuclear reprogramming is a process of transforming one cell type into an unrelated cell type by epigenetic changes that alter the cell’s original gene expression pattern. Such epigenetic changes force cells to express a different set of genes, which play a significant role in inducing transformation into other cell types. Nuclear reprogramming offers applications in reproductive cloning for livestock propagation and regenerative medicine — developing patient-specific cells for...
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CRISPR and crRNAs02:53

CRISPR and crRNAs

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Bacteria and archaea are susceptible to viral infections just like eukaryotes; therefore, they have developed a unique adaptive immune system to protect themselves. Clustered regularly interspaced short palindromic repeats and CRISPR-associated proteins (CRISPR-Cas) are present in more than 45% of known bacteria and 90% of known archaea.
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Related Experiment Video

Updated: Jun 17, 2025

In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing
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In Vitro Selection of Engineered Transcriptional Repressors for Targeted Epigenetic Silencing

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Precision epigenetic editing: Technological advances, enduring challenges, and therapeutic applications.

Goldie V Roth1, Isabella R Gengaro2, Lei S Qi3

  • 1Department of Chemical Engineering, Stanford University, Stanford, CA, USA.

Cell Chemical Biology
|August 13, 2024
PubMed
Summary

Epigenetic editing tools precisely control gene expression for therapeutic purposes. Future advancements in engineering and delivery technologies will enable safe and effective treatment of diverse disorders.

Keywords:
CRISPRchromatin modificationschromatin reorganizationdeliveryepigenetic diseaseepigenetic editingepigeneticsepigenome therapyneurological disease

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Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
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Area of Science:

  • Molecular Biology
  • Genetics
  • Epigenetics

Background:

  • The epigenome modulates gene expression for cellular processes like aging and disease.
  • Understanding and controlling the epigenome is crucial for therapeutic interventions.

Purpose of the Study:

  • To review current targeted epigenetic editing tools.
  • To discuss technical considerations and future development opportunities.
  • To explore therapeutic applications and delivery challenges.

Main Methods:

  • Review of existing epigenetic editing technologies.
  • Analysis of technical aspects and engineering tools.
  • Discussion of therapeutic applications and delivery systems.

Main Results:

  • A diverse toolbox of epigenetic editing technologies is available.
  • Engineering tools and delivery technologies are advancing rapidly.
  • Delivery challenges, especially for brain interventions, are being addressed.

Conclusions:

  • Epigenetic editing holds significant therapeutic potential for various diseases.
  • Continued advancements in engineering and delivery are key to clinical success.
  • Epigenetic editing is poised to become a powerful treatment modality across all tissues.