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Related Concept Videos

Transgenic Organisms00:53

Transgenic Organisms

Overview
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.
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...
Tumor Progression02:07

Tumor Progression

Tumor progression is a phenomenon where the pre-formed tumor acquires successive mutations to become clinically more aggressive and malignant. In the 1950s, Foulds first described the stepwise progression of cancer cells through successive stages.
Colon cancer is one of the best-documented examples of tumor progression. Early mutation in the APC gene in colon cells causes a small growth on the colon wall called a polyp. With time, this polyp grows into a benign, pre-cancerous tumor. Further...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...

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Gene therapy progress and prospects: targeted gene repair.

Gene therapy·2005
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Gene editing of a human gene in yeast artificial chromosomes using modified single-stranded DNA and dual targeting.

The pharmacogenomics journal·2004
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Targeted gene repair in mammalian cells using chimeric RNA/DNA oligonucleotides and modified single-stranded vectors.

Science's STKE : signal transduction knowledge environment·2001
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In vivo gene repair of point and frameshift mutations directed by chimeric RNA/DNA oligonucleotides and modified single-stranded oligonucleotides.

Nucleic acids research·2001
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Chloroplast lysates support directed mutagenesis via modified DNA and chimeric RNA/DNA oligonucleotides.

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Genetic Manipulation in Δku80 Strains for Functional Genomic Analysis of Toxoplasma gondii
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Genetic Manipulation in Δku80 Strains for Functional Genomic Analysis of Toxoplasma gondii

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Progress and prospects: targeted gene alteration (TGA).

H Parekh-Olmedo1, E B Kmiec

  • 1Department of Biological Sciences, Delaware Biotechnology Institute, University of Delaware, Newark, DE 19711, USA.

Gene Therapy
|November 2, 2007
PubMed
Summary

Targeted gene repair uses DNA oligonucleotides to correct mutations causing genetic diseases. Recent advances show clinical potential but also highlight new challenges for this gene editing technology.

Area of Science:

  • Molecular biology
  • Genetics
  • Biotechnology

Background:

  • Genetic diseases are often caused by single base mutations.
  • Targeted gene repair aims to correct these mutations at the DNA level.

Purpose of the Study:

  • To review recent advances in targeted gene repair technology.
  • To assess the clinical potential and challenges of gene editing for genetic diseases.

Main Methods:

  • Utilizing single-stranded DNA oligonucleotides to guide nucleotide exchange.
  • Investigating the mechanisms of mutation correction in genomic sites.

Main Results:

  • Significant progress in understanding gene repair mechanisms.
  • Identification of viable clinical targets for gene editing.

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Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
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Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

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Last Updated: Jul 10, 2026

Genetic Manipulation in Δku80 Strains for Functional Genomic Analysis of Toxoplasma gondii
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Genetic Manipulation in Δku80 Strains for Functional Genomic Analysis of Toxoplasma gondii

Published on: July 12, 2013

Mosaic Zebrafish Transgenesis for Functional Genomic Analysis of Candidate Cooperative Genes in Tumor Pathogenesis
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Mosaic Zebrafish Transgenesis for Functional Genomic Analysis of Candidate Cooperative Genes in Tumor Pathogenesis

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Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
09:37

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

Published on: August 25, 2021

  • Transition from basic science to translational research.

    • Conclusions:

    • Targeted gene repair technology is advancing towards clinical applications.
    • Recent developments offer promise but also introduce new considerations for safety and efficacy.