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Related Concept Videos

Parkinson's Disease: Treatment01:24

Parkinson's Disease: Treatment

Neurodegenerative disorders, such as Parkinson's Disease (PD), involve the gradual and irreversible destruction of neurons in particular brain areas. These disorders exhibit standard features like proteinopathies, selective vulnerability of some neurons, and an interaction of intrinsic properties, genetics, and environmental influences in neural injury.
Parkinson's Disease is primarily a result of the loss of dopaminergic neurons in the substantia nigra pars compacta. The cornerstone of its...
Parkinson Disease ll: Pathophysiology01:24

Parkinson Disease ll: Pathophysiology

Parkinson disease (PD) is a progressive neurodegenerative disorder primarily affecting movement, with additional non-motor features. Its pathophysiology involves complex interactions among genetic susceptibility, environmental exposures, and cellular dysfunction, including dopaminergic neuron loss, protein aggregation, and mitochondrial impairment.Selective NeurodegenerationA key feature is the degeneration of dopaminergic neurons in the substantia nigra pars compacta, leading to reduced...
Parkinson Disease l: Introduction01:24

Parkinson Disease l: Introduction

Parkinson’s disease is a chronic, progressive neurodegenerative disorder that primarily affects movement. It is characterized by motor symptoms such as resting tremors, muscle rigidity, bradykinesia (slowness of movement), and postural instability. Patients may notice hand tremors at rest, stiffness during movement, or a shuffling gait. In addition to motor features, non-motor symptoms include sleep disturbances, mood and behavioral changes, constipation, and cognitive impairment, all of which...
Parkinson's Disease: Overview01:15

Parkinson's Disease: Overview

Neurodegenerative disorders are progressive diseases that cause irreversible damage and loss to neurons in specific brain areas. Examples of these disorders include Parkinson's disease, Alzheimer's disease, Multiple Sclerosis (MS), and Amyotrophic Lateral Sclerosis (ALS). These disorders share characteristics such as proteinopathies, selective neuronal vulnerability, and a complex interplay between genetic and environmental factors. The primary therapeutic goal for these conditions is to...
Epigenetic Regulation01:46

Epigenetic Regulation

Epigenetic mechanisms play an essential role in healthy development. Conversely, precisely regulated epigenetic mechanisms are disrupted in diseases like cancer.
Epigenetic Regulation01:37

Epigenetic Regulation

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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Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models
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Epigenetic engineering to reverse the Parkinson's expression state.

André X C N Valente1, Ricardo Pires das Neves, Paulo J Oliveira

  • 1Systems Biology Group, Biocant - Biotechnology Innovation Center, Cantanhede, Portugal. andre.valente@biocant.pt

Parkinsonism & Related Disorders
|May 15, 2012
PubMed
Summary

Researchers propose a new approach to treat sporadic Parkinson's disease by epigenetically reprogramming cells. This method aims to reverse the pathological PD-state by targeting mitochondrial dysfunction, offering a potential new therapeutic avenue.

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Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases

Published on: May 2, 2025

Area of Science:

  • Neuroscience
  • Cell Biology
  • Genetics

Background:

  • Sporadic Parkinson's disease (PD) affects millions globally, with its origins and cellular propagation remaining unclear.
  • Current therapeutic strategies are limited due to the unknown nature of PD initiation.
  • The study posits that sporadic PD represents a pathological cellular state (PD-state) that may be epigenetically induced.

Purpose of the Study:

  • To propose an in vitro high-throughput screening method for identifying molecular cocktails.
  • To investigate the potential for epigenetic reprogramming to reverse the cellular PD-state.
  • To explore mitochondrial bioenergetics as a key phenotype of the PD-state and a target for therapeutic intervention.

Main Methods:

  • Developing an in vitro screening assay to search for epigenetic reprogramming molecules.
  • Utilizing a metabolic challenge as a preliminary screen to assess the reversal of the PD-state.
  • Measuring improvements in cellular energy metabolism as an indicator of PD-state reversal.

Main Results:

  • A generic multi-tissue PD-state phenotype is characterized by defects in mitochondrial bioenergetics.
  • The proposed method allows for a high-throughput search for epigenetic reprogramming agents.
  • Metabolic improvement serves as a viable preliminary screen for assessing PD-state reversal.

Conclusions:

  • Epigenetic reprogramming offers a theoretical possibility for reverting the PD-state to normality.
  • Targeting mitochondrial dysfunction through metabolic interventions may be a key therapeutic strategy for sporadic PD.
  • The proposed screening approach could accelerate the discovery of novel treatments for Parkinson's disease.