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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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Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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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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What is Genetic Engineering?00:49

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Overview
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CRISPR01:59

CRISPR

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against...
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In-vitro Mutagenesis01:16

In-vitro Mutagenesis

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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.
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Related Experiment Video

Updated: Dec 19, 2025

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

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A primer to gene therapy: Progress, prospects, and problems.

Hidde A Zittersteijn1, Manuel A F V Gonçalves1, Rob C Hoeben1

  • 1Department of Cell and Chemical Biology, Leiden University Medical Center, Leiden, The Netherlands.

Journal of Inherited Metabolic Disease
|June 9, 2020
PubMed
Summary

Gene therapies using viral vectors offer new hope for genetic disorders. Advances in gene editing and regulatory development are crucial for translating these technologies into approved advanced therapy medicinal products.

Keywords:
advanced therapy medicinal productsgene editinggenetic diseasegenetic therapyprogrammable nucleasesviral vectors

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Area of Science:

  • * Genetics and Molecular Biology
  • * Pharmacology and Therapeutics

Background:

  • * Gene addition therapies have achieved clinical success, with approved products available.
  • * Gene editing technologies, including CRISPR-Cas9, offer direct correction of disease-causing mutations in somatic cells.
  • * Viral vector-based gene delivery is central to implementing genetic therapies for inherited and acquired disorders.

Purpose of the Study:

  • * To review state-of-the-art viral vector systems and gene-centered therapies.
  • * To summarize emerging gene editing approaches like CRISPR-Cas9, nickases, base editors, and prime editors.
  • * To highlight regulatory considerations for developing advanced therapy medicinal products.

Main Methods:

  • * Review of current literature on viral vector gene delivery systems.
  • * Summary of gene editing technologies and their applications.
  • * Analysis of regulatory aspects for advanced therapy medicinal products.

Main Results:

  • * Viral vectors are key for delivering gene therapies, particularly for inborn metabolic disorders.
  • * Gene editing tools are rapidly evolving, expanding therapeutic possibilities.
  • * Technological advancements in gene therapy are outpacing regulatory frameworks.

Conclusions:

  • * Viral vector gene therapy shows significant promise for treating genetic diseases.
  • * Gene editing offers precise correction of genetic defects, advancing therapeutic options.
  • * Further regulatory development is essential to facilitate the clinical translation of gene therapies and gene editing into approved advanced therapy medicinal products (ATMPs).