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Gene Therapy

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...

Reporter Genes

Reporter Genes

Reporter genes are a type of protein-coding gene that are often tagged to a gene of interest. Once inside a target cell, reporter genes usually produce visually identifiable characteristics like fluorescence and luminescence when expressed along with the gene of interest. Thus, reporter genes “report” the presence or absence of genes of interest in an organism, determine the gene expression pattern, or track the physical location of a DNA segment or protein in the cell.

Conservative Site-specific Recombination and Phase Variation

Conservative Site-specific Recombination and Phase Variation

Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...

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Related Experiment Video

Updated: Nov 6, 2025

Electroporation of Plasmid DNA into Mouse Skeletal Muscle

Electroporation of Plasmid DNA into Mouse Skeletal Muscle

Published on: April 6, 2022

Muscle-Specific Promoters for Gene Therapy.

V V Skopenkova^1,2,3, T V Egorova^1,2, M V Bardina^1,2,3

¹Institute of Gene Biology, Russian Academy of Sciences, Moscow, 119334 Russia.

|May 7, 2021

Summary

This summary is machine-generated.

Developing effective gene therapies for muscular disorders requires precise control. This review highlights advancements in muscle-specific promoters for robust transgene expression in target tissues, crucial for gene replacement therapy success.

Keywords:

AAV Gene therapy muscle-specific promoters natural promoters synthetic promoters

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Last Updated: Nov 6, 2025

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Transplantation of Induced Pluripotent Stem Cell-derived Mesoangioblast-like Myogenic Progenitors in Mouse Models of Muscle Regeneration

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Isolation and Immortalization of Patient-derived Cell Lines from Muscle Biopsy for Disease Modeling

Isolation and Immortalization of Patient-derived Cell Lines from Muscle Biopsy for Disease Modeling

Published on: January 18, 2015

Area of Science:

Biomedical Engineering
Molecular Biology
Genetics

Background:

Genetic diseases cause numerous muscular disorders, necessitating advanced treatments.
Gene replacement therapy offers a promising strategy by delivering functional genes via viral vectors.
Effective therapy requires precise control over gene expression in target muscles.

Purpose of the Study:

To review the development and optimization of muscle-specific promoters for gene therapy.
To explore synthetic promoter engineering and viral vector components for tissue-specific expression.
To provide current information on clinical and preclinical gene therapy trials using these promoters.

Main Methods:

Focus on muscle-specific promoters derived from skeletal muscle α-actin, muscle creatine kinase, and desmin genes.
Analysis of current approaches for engineering synthetic muscle-specific promoters.
Discussion of viral vector elements influencing tissue-specific gene delivery.

Main Results:

Significant progress has been made in designing regulatory sequences for high, robust transgene expression in skeletal muscle, diaphragm, and heart.
Engineered promoters demonstrate limited activity in non-target tissues, enhancing safety.
Up-to-date information on clinical and preclinical trials is presented.

Conclusions:

Muscle-specific promoters are critical for the efficacy and safety of gene replacement therapies for muscular disorders.
Continued development of these promoters and viral vectors will advance gene therapy applications.
The reviewed information supports the ongoing progress in clinical gene therapy for muscle diseases.