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Related Concept Videos

Gene Therapy00:59

Gene Therapy

27.6K
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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Mismatch Repair01:36

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Mismatch Repair01:20

Mismatch Repair

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Organisms are capable of detecting and fixing nucleotide mismatches that occur during DNA replication. This sophisticated process requires identifying the new strand and replacing the erroneous bases with correct nucleotides. Mismatch repair is coordinated by many proteins in both prokaryotes and eukaryotes.
The Mutator Protein Family Plays a Key Role in DNA Mismatch Repair
The human genome has more than 3 billion base pairs of DNA per cell. Prior to cell division, that vast amount of genetic...
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Overview of DNA Repair02:25

Overview of DNA Repair

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In order to be passed through generations, genomic DNA must be undamaged and error-free. However, every day, DNA in a cell undergoes several thousand to a million damaging events by natural causes and external factors. Ionizing radiation such as UV rays, free radicals produced during cellular respiration, and hydrolytic damage from metabolic reactions can alter the structure of DNA. Damages caused include single-base alteration, base dimerization, chain breaks, and cross-linkage.
Chemically...
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Base Excision Repair01:54

Base Excision Repair

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One of the common DNA damages is the chemical alteration of single bases by alkylation, oxidation, or deamination. The altered bases cause mispairing and strand breakage during replication. This type of damage causes minimal change to the DNA double helix structure and can be repaired by the base excision repair (BER) pathways. BER corrects damaged DNA sequences by removing the damaged base and restoring the original base sequence using the complementary strand as a template.
The first step of...
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Nucleotide Excision Repair01:08

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Related Experiment Video

Updated: Jan 31, 2026

Production and Purification of Baculovirus for Gene Therapy Application
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Production and Purification of Baculovirus for Gene Therapy Application

Published on: April 9, 2018

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Repairing the Brain: Gene Therapy.

Tomas Björklund1

  • 1Molecular Neuromodulation, Wallenberg Neuroscience Center, Lund University, Lund, Sweden.

Journal of Parkinson'S Disease
|December 26, 2018
PubMed
Summary

Gene therapy for neurodegenerative disorders, especially Parkinson's disease, faces challenges but is advancing. Future clinical applications are expected to become more common within two decades.

Area of Science:

  • Neuroscience
  • Genetics
  • Clinical Medicine

Background:

  • In vivo gene therapy for neurodegenerative disorders presents significant challenges.
  • Despite being a relatively young field (around 20 years), clinical translation has been slower than anticipated.
  • Parkinson's disease patients have experienced prolonged waits for effective gene therapy treatments.

Purpose of the Study:

  • To review the challenges and progress in in vivo gene therapy for neurodegenerative disorders.
  • To discuss the scientific and economic factors hindering clinical application.
  • To project the future role of gene therapy in treating Parkinson's disease and other neurological conditions.

Main Methods:

  • Review of pre-clinical proof-of-concept studies.
Keywords:
Genetic therapyParkinson’s diseaseclinical trialdependovirusgene editingneuroprotectionrejuvenation

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  • Analysis of scientific and economic barriers.
  • Forecasting future trends in gene therapy development and clinical integration.

    • Main Results:

    • Gene therapy for neurodegenerative diseases has encountered numerous scientific and economic hurdles.
    • Many promising therapies have not progressed beyond early-stage research.
    • Progress is being made, indicating a more optimistic outlook for the next 20 years.

    Conclusions:

    • In vivo gene therapy for neurodegenerative disorders, including Parkinson's disease, is a complex but evolving field.
    • Overcoming scientific and economic obstacles is crucial for clinical success.
    • Gene therapy is anticipated to become an integral part of future clinical treatments for Parkinson's disease.