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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...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Gene Therapy00:59

Gene Therapy

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 inserted. The...
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...
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...

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Related Experiment Video

Updated: May 16, 2026

Gene-environment Interaction Models to Unmask Susceptibility Mechanisms in Parkinson's Disease
08:09

Gene-environment Interaction Models to Unmask Susceptibility Mechanisms in Parkinson's Disease

Published on: January 7, 2014

[Gene therapy for Parkinson's disease].

Shin-Ichi Muramatsu1

  • 1Division of Neurology, Department of Medicine, Jichi Medical University.

Rinsho Shinkeigaku = Clinical Neurology
|December 1, 2012
PubMed
Summary

Gene therapy using adeno-associated virus (AAV) vectors to deliver the aromatic (L)-amino acid decarboxylase (AADC) gene shows promise for Parkinson's disease (PD). This approach improved motor function in PD patients, suggesting potential benefits for advanced disease.

Area of Science:

  • Neurology
  • Gene Therapy
  • Molecular Neuroscience

Context:

  • Parkinson's disease (PD) is a progressive neurodegenerative disorder.
  • Current treatments aim to manage symptoms but do not halt disease progression.
  • Gene therapy offers a novel strategy to address the underlying pathology of PD.

Purpose:

  • To evaluate the safety and efficacy of adeno-associated virus (AAV) vector-mediated gene transfer of aromatic (L)-amino acid decarboxylase (AADC) in Parkinson's disease patients.
  • To assess the long-term motor outcomes in patients receiving intra-putaminal AADC gene therapy.

Summary:

  • A phase I clinical trial investigated the safety of intra-putaminal AAV vector infusion encoding AADC in six advanced PD patients.
  • Patients demonstrated sustained improvements in motor scores on the Unified Parkinson's Disease Rating Scale (UPDRS) at 36 months post-treatment.

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Ole Isacson: Development of New Therapies for Parkinson's Disease
23:53

Ole Isacson: Development of New Therapies for Parkinson's Disease

Published on: April 29, 2007

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Last Updated: May 16, 2026

Gene-environment Interaction Models to Unmask Susceptibility Mechanisms in Parkinson's Disease
08:09

Gene-environment Interaction Models to Unmask Susceptibility Mechanisms in Parkinson's Disease

Published on: January 7, 2014

Ole Isacson: Development of New Therapies for Parkinson's Disease
23:53

Ole Isacson: Development of New Therapies for Parkinson's Disease

Published on: April 29, 2007

  • The study also noted symptom amelioration in children with AADC deficiency treated with a similar AAV vector approach.

    • Impact:

    • AAV-mediated AADC gene transfer shows encouraging efficacy for advanced Parkinson's disease.
    • This gene therapy strategy may offer a new therapeutic avenue for PD patients.
    • The findings support further investigation into gene therapy for neurodegenerative disorders.