Related Experiment Video

Updated: Jun 19, 2026

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
13:47

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

Published on: March 29, 2019

Transcription factors as therapeutic targets in CNS disorders

Michael J Kane1, Bruce A Citron

  • 1Laboratory of Molecular Biology, Research & Development 151, Bay Pines VA Healthcare System and Department of Molecular Medicine, University of South Florida College of Medicine, Bay Pines, FL 33744, USA.

Recent Patents on CNS Drug Discovery
|November 7, 2009
PubMed

Insights

Programmed neuronal loss in CNS disorders is linked to altered gene expression. Targeting pro- and anti-apoptotic transcription factors offers a therapeutic window to prevent cell death and neurodegeneration.

Area of Science:

  • Neuroscience
  • Molecular Biology
  • Genetics

Background:

  • Central nervous system (CNS) disorders often involve programmed neuronal loss.
  • Neurodegenerative diseases, traumatic, and ischemic injuries alter gene expression impacting cell survival.
  • Mechanisms driving altered gene expression in CNS cell death remain incompletely understood.

Purpose of the Study:

  • To review recent patents related to modulating cell death factors in CNS disorders.
  • To explore the role of five key transcription factors in neuronal survival and death.
  • To highlight the therapeutic potential of targeting transcription factor dysregulation.

Main Methods:

  • Literature review of recent patents.
  • Analysis of gene expression changes in CNS disorders.
  • Focus on pro- and anti-apoptotic transcription factors.

Main Results:

  • Dysregulation of transcription factors is an early event in CNS disorders.
  • Altered balance of transcription factors induces pro-cell death gene expression.
  • Patents aim to modulate these factors to prevent or mitigate neuronal damage.

Conclusions:

  • Targeting transcription factors presents a therapeutic opportunity for CNS disorders.
  • Intervention may be most effective within a narrow window before irreversible neuronal death.
  • Further research into these factors could improve outcomes for affected individuals.

More Related Videos

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes10:33

Delivery of Therapeutic siRNA to the CNS Using Cationic and Anionic Liposomes

Published on: July 23, 2016

Related Concept Videos

Transcription Factors02:16

Transcription Factors

Tissue-specific transcription factors contribute to diverse cellular functions in mammals. For example, the gene for beta globin, a major component of hemoglobin, is present in all cells of the body. However, it is only expressed in red blood cells because the transcription factors that can bind to the promoter sequences of the beta globin gene are only expressed in these cells. Tissue-specific transcription factors also ensure that mutations in these factors may impair only the function of...
Transcription Factors02:16

Transcription Factors

Tissue-specific transcription factors contribute to diverse cellular functions in mammals. For example, the gene for beta globin, a major component of hemoglobin, is present in all cells of the body. However, it is only expressed in red blood cells because the transcription factors that can bind to the promoter sequences of the beta globin gene are only expressed in these cells. Tissue-specific transcription factors also ensure that mutations in these factors may impair only the function of...
General Transcription Factors01:30

General Transcription Factors

Tissue-specific transcription factors contribute to diverse cellular functions in mammals. For example, the gene for beta globin, a major component of hemoglobin, is present in all cells of the body. However, it is only expressed in red blood cells because the transcription factors that can bind to the promoter sequences of the beta globin gene are only expressed in these cells. Tissue-specific transcription factors also ensure that mutations in these factors may impair only the function of...
Eukaryotic Transcription Inhibitors01:52

Eukaryotic Transcription Inhibitors

Certain biochemical processes, such as embryonic development and cell growth regulation, depend on the repression of specific genes. DNA binding proteins known as eukaryotic transcription inhibitors regulate the repression of gene expression in eukaryotes. The presence of these inhibitors at the required location and time in the cell is triggered by the presence of hormones and additional signals from other cells.
Eukaryotic transcription inhibitors usually contain two distinct domains, a DNA...
Master Transcription Regulators02:23

Master Transcription Regulators

Master transcription regulators are regulatory proteins that are predominantly responsible for regulating the expression of multiple genes. Often these genes work in concert to drive a  complex process. Activation of a master transcription regulator can lead to a cascade of transcriptional activation necessary for that outcome. These regulators can directly bind to the regulatory sequences of the various genes involved, or they can indirectly regulate transcription by binding to regulatory...
Eukaryotic Transcription Activators02:42

Eukaryotic Transcription Activators

Transcription activators are proteins that promote the transcription of genes from DNA to RNA. In most cases, these proteins contain two separate domains ‒ a domain that binds to DNA and a domain for activating transcription; however, in some cases, a single domain is responsible for both binding and activation of transcription, as seen in the glucocorticoid receptor and MyoD.
The binding domains are capable of recognizing and interacting with regulatory sequences on the DNA. These domains are...