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Related Concept Videos

Epigenetic Regulation01:46

Epigenetic Regulation

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Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.
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Chromatin Modification in iPS Cells01:32

Chromatin Modification in iPS Cells

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Chromatin modification alters gene expression; therefore, scientists can add histone-modifying enzymes, histone variants, and chromatin remodeling complexes to somatic cells to aid reprogramming into pluripotent stem (iPS) cells.
Compact chromatin makes reprogramming difficult. Enzymes, such as histone demethylases and acetyltransferases, are often added during reprogramming to loosen the chromatin, making the DNA more accessible to transcription factors. Molecules that inhibit histone...
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Methods of Nuclear Reprogramming01:24

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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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Gene Therapy00:59

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Genomic Imprinting and Inheritance02:30

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Diploid organisms inherit genetic material through chromosomes from both parents. Copies of the same gene are known as alleles. In most cases, both alleles are simultaneously expressed and allow various cellular processes to function optimally. If one of the alleles is missing or mutated, the expression of the other allele can compensate; however, this is not true for all genes.
The expression of some genes depends on which parent passed the gene to the offspring, through a phenomenon known as...
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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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Related Experiment Video

Updated: Jun 4, 2025

Repressing Gene Transcription by Redirecting Cellular Machinery with Chemical Epigenetic Modifiers
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Repressing Gene Transcription by Redirecting Cellular Machinery with Chemical Epigenetic Modifiers

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Epigenetic Therapies.

Wallace Bourgeois1, Scott A Armstrong1, Emily B Heikamp2

  • 1Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, and Harvard Medical School, Boston, Massachusetts 02215, USA.

Cold Spring Harbor Perspectives in Medicine
|December 18, 2024
PubMed
Summary
This summary is machine-generated.

Epigenetic therapies offer new hope for pediatric cancers by targeting abnormal gene regulation. These treatments aim to reverse epigenetic changes, potentially restoring normal cell development and fighting childhood cancers.

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Area of Science:

  • Pediatric Oncology
  • Epigenetics
  • Cancer Therapeutics

Background:

  • Pediatric tumors often exhibit epigenetic dysregulation and impaired differentiation, rather than high mutation rates.
  • Epigenetic alterations, including DNA methylation and histone modifications, play a crucial role in childhood cancer development.
  • Targeting epigenetic regulators offers a promising therapeutic strategy for pediatric malignancies.

Purpose of the Study:

  • To review emerging epigenetic therapies for pediatric cancers.
  • To highlight the mechanisms of action for current and investigational epigenetic drugs.
  • To discuss the potential of targeting epigenetic dysregulation in childhood tumors.

Main Methods:

  • Review of current literature on epigenetic therapies in pediatric oncology.
  • Analysis of FDA-approved epigenetic drugs and their mechanisms (e.g., DNA methyltransferase inhibitors, IDH inhibitors).
  • Discussion of emerging strategies targeting histone modifications and oncogenic fusion proteins.

Main Results:

  • DNA hypomethylating agents (azacitidine, decitabine) are FDA-approved for hematologic malignancies.
  • IDH inhibitors are approved for certain hematologic and solid tumors with specific mutations.
  • Small molecules targeting histone-modifying enzymes are under investigation for pediatric tumors.
  • Therapies disrupting oncogenic fusion protein-chromatin complexes show significant promise, with established use in some leukemias.

Conclusions:

  • Epigenetic therapies represent a rapidly advancing frontier in pediatric cancer treatment.
  • Targeting DNA methylation, histone modifications, and fusion proteins offers diverse therapeutic avenues.
  • Further understanding of epigenetic mechanisms in pediatric cancers will expand the landscape of druggable targets.