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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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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Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy01:30

Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy

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Various diagnostic tests are employed in the diagnostic process for Inflammatory Bowel Disease (IBD), particularly to differentiate between Crohn's disease and ulcerative colitis.
Diagnostic studies
A colonoscopy is the definitive screening test, distinguishing ulcerative colitis from other colon diseases with similar symptoms. During a colonoscopy test, inflamed mucosa with exudate ulcerations can be observed, and biopsies are taken to determine the histologic characteristics of the...
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Group Therapy01:26

Group Therapy

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Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...
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Gene Flow02:39

Gene Flow

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Gene flow is the transfer of genes among populations, resulting from either the dispersal of gametes or from the migration of individuals.
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Gene Families01:57

Gene Families

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Gene families consist of groups of genes proposed to have originated from a common ancestor. Typically these arise through events in which a gene or genes are mistakenly duplicated during cell division. Unlike their parent genes (which are subject to selection pressure to maintain function), these gene copies do not need to preserve their sequences and may evolve at a relatively faster rate.
Occasionally these regions can be adapted to take on new roles within the organism, becoming novel genes...
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Gene Conversion02:08

Gene Conversion

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Other than maintaining genome stability via DNA repair, homologous recombination plays an important role in diversifying the genome. In fact, the recombination of sequences forms the molecular basis of genomic evolution. Random and non-random permutations of genomic sequences create a library of new amalgamated sequences. These newly formed genomes can determine the fitness and survival of cells. In bacteria, homologous and non-homologous types of recombination lead to the evolution of new...
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Related Experiment Video

Updated: Feb 14, 2026

Ole Isacson: Development of New Therapies for Parkinson's Disease
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Gene Therapies for Polyglutamine Diseases.

Carlos A Matos1,2, Vítor Carmona1,3, Udaya-Geetha Vijayakumar1

  • 1CNC-Center for Neuroscience and Cell Biology, University of Coimbra, Coimbra, Portugal.

Advances in Experimental Medicine and Biology
|February 11, 2018
PubMed
Summary

Gene therapy offers new hope for untreatable polyglutamine diseases by targeting the genetic cause. Research focuses on effective delivery systems and molecular strategies to counteract neurodegeneration.

Keywords:
Gene silencingGene therapyPolyglutamine diseasesRNA interferenceViral and non-viral vectors

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Area of Science:

  • Neuroscience
  • Genetics
  • Molecular Biology

Background:

  • Polyglutamine diseases are inherited neurological disorders currently lacking effective treatments.
  • Understanding the molecular basis and advancing gene therapy tools are key to developing potential therapies.

Purpose of the Study:

  • To review gene therapy strategies for polyglutamine diseases, focusing on nucleic acid delivery systems and therapeutic agent actions.
  • To explore the potential of various gene-targeting approaches.

Main Methods:

  • Review of viral and non-viral vector systems for central nervous system gene delivery.
  • Investigation of RNA interference (RNAi) strategies, including small interfering RNAs (siRNAs), short hairpin RNAs (shRNAs), and microRNAs (miRNAs).
  • Evaluation of gene overexpression and gene editing tools for therapeutic intervention.

Main Results:

  • Both viral and non-viral vectors show promise for CNS gene delivery, each with distinct advantages and limitations.
  • RNAi-based strategies effectively target disease-causing genes by silencing protein products.
  • Gene editing technologies offer upstream intervention possibilities.

Conclusions:

  • Gene therapy presents a promising therapeutic avenue for polyglutamine diseases.
  • Successful preclinical results in cell and animal models support further development and future clinical application.