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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

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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.
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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

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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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Cell Specific Gene Expression01:58

Cell Specific Gene Expression

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Multicellular organisms contain a variety of structurally and functionally distinct cell types, but the DNA in all the cells originated from the same parent cells. The differences in the cells can be attributed to the differential gene expression. Liver cells, whose functions include detoxification of blood, production of bile to metabolize fats, and synthesis of proteins essential for metabolism, must express a specific set of genes to perform their functions. Gene expression also varies with...
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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.
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Related Experiment Video

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Ole Isacson: Development of New Therapies for Parkinson's Disease
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Gene Therapy Tools for Brain Diseases.

Selene Ingusci1, Gianluca Verlengia1,2, Marie Soukupova1

  • 1Department of Medical Sciences and National Institute of Neuroscience, University of Ferrara, Ferrara, Italy.

Frontiers in Pharmacology
|July 18, 2019
PubMed
Summary

Gene therapy offers a promising approach for treating complex central nervous system (CNS) disorders by delivering therapeutic genes. This review explores viral and non-viral vectors for CNS gene delivery and advanced genetic regulation techniques.

Keywords:
brain diseasecentral nervous systemgene regulationgene therapyviral vector

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Area of Science:

  • Neuroscience
  • Molecular Biology
  • Biotechnology

Background:

  • Central nervous system (CNS) disorders are challenging to treat due to complexity and the blood-brain barrier.
  • Current treatments for neurological diseases are often insufficient, lacking specificity and effectiveness.
  • Gene therapy presents a viable strategy for delivering therapeutic molecules to the CNS.

Purpose of the Study:

  • To review current gene therapy tools for the central nervous system.
  • To discuss viral and non-viral vectors, their applications, and limitations.
  • To explore advanced genetic regulation strategies for CNS gene therapy.

Main Methods:

  • Review of literature on gene delivery vectors for the CNS.
  • Analysis of viral vectors (e.g., herpes simplex virus type 1) and non-viral methods.
  • Examination of genetic regulation techniques including promoters, RNA interference, optogenetics, DREADDs, and gene editing.

Main Results:

  • Viral and non-viral vectors are key tools for CNS gene delivery.
  • Cell-specific and inducible promoters enable targeted gene expression.
  • Advanced systems like optogenetics, DREADDs, and gene editing offer precise control over gene function.

Conclusions:

  • Gene therapy holds significant potential for treating neurological disorders.
  • Herpes simplex virus type 1-derived vectors are highlighted for simultaneous multi-gene delivery.
  • Precise control over gene expression is crucial for effective and safe CNS therapies.