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Related Concept Videos

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...
Gene Evolution - Fast or Slow?02:05

Gene Evolution - Fast or Slow?

The genomes of eukaryotes are punctuated by long stretches of sequence which do not code for proteins or RNAs. Although some of these regions do contain crucial regulatory sequences, the vast majority of this DNA serves no known function. Typically, these regions of the genome are the ones in which the fastest change, in evolutionary terms, is observed, because there is typically little to no selection pressure acting on these regions to preserve their sequences.
In contrast, regions which code...
Gene Evolution - Fast or Slow?02:05

Gene Evolution - Fast or Slow?

The genomes of eukaryotes are punctuated by long stretches of sequence which do not code for proteins or RNAs. Although some of these regions do contain crucial regulatory sequences, the vast majority of this DNA serves no known function. Typically, these regions of the genome are the ones in which the fastest change, in evolutionary terms, is observed, because there is typically little to no selection pressure acting on these regions to preserve their sequences.
In contrast, regions which code...
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

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Related Experiment Video

Updated: May 14, 2026

Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun
06:40

Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun

Published on: October 24, 2014

Gene therapy: science in slow motion.

John Carroll

    Biotechnology Healthcare
    |February 12, 2013
    PubMed
    Summary
    This summary is machine-generated.

    Early-stage gene therapy trials show promise for deadly diseases. Despite past setbacks, researchers are optimistic about attracting biotech investment for future development.

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    Published on: December 17, 2013

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    Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun
    06:40

    Regioselective Biolistic Targeting in Organotypic Brain Slices Using a Modified Gene Gun

    Published on: October 24, 2014

    In vivo Reprogramming of Adult Somatic Cells to Pluripotency by Overexpression of Yamanaka Factors
    12:12

    In vivo Reprogramming of Adult Somatic Cells to Pluripotency by Overexpression of Yamanaka Factors

    Published on: December 17, 2013

    Area of Science:

    • Biotechnology
    • Genetics
    • Medical Research

    Background:

    • Gene therapy offers potential treatments for severe diseases.
    • Past challenges in the field have impacted investor confidence.
    • Current research aims to overcome previous limitations.

    Purpose of the Study:

    • To evaluate the progress of early-stage human gene therapy trials.
    • To assess the potential for gene therapy to address major global health threats.
    • To understand investor sentiment and its influence on biotech company involvement.

    Main Methods:

    • Monitoring ongoing early-stage human clinical trials.
    • Analyzing historical data on gene therapy setbacks and successes.
    • Gauging researcher and investor outlook on the field's future.

    Main Results:

    • Early human trials are actively progressing.
    • Optimism exists regarding the therapeutic potential of gene therapies.
    • Investor caution persists due to prior challenges.

    Conclusions:

    • Gene therapy research is advancing, with early trials underway.
    • Successful outcomes could revitalize biotech investment in the sector.
    • The field holds significant promise for treating intractable diseases.