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What Is Gene Editing?

The development of genetic engineering technology has being boomed since the first targeted gene alteration by homologous recombination was produced in the 1970s. Gene editing, also known as genome editing, is a new kind of gene engineering technology that accurately modifies the specific target gene of the living organism genome. Different from earlier gene engineering technology of randomly inserting exogenous genetic material into the host genome, gene editing enables efficient gene addition, deletion, modification or alteration at particular locations in the genome.

What Does Gene Editing Actually "Edit"?

Gene editing is a powerful and promising tool for gene therapy of various diseases. The ultimate goal of gene editing is to produce temporary or permanent therapeutic nucleic acids that can be expressed normally and stably, which is also an essential step of gene therapy. By introducing therapeutic genes (including protein-coding genes and non-coding nucleic acids) into the host chromosome, or by removal/replacement of pathogenic genes, some hereditary disorders, cancers, and other diseases can be effectively treated by gene therapy.

How to Achieve Genome Editing?

Conventional gene editing techniques, such as homologous recombination and gene targeting, are hindered by their low efficiency and poor adaptability of species. With the development of gene editing technology and the emergence of various biotechnologies, rapid and economical specific modifications can be introduced into a wide range of cell types and organism genomes. Currently, the most commonly used approaches for gene editing are:

1. Transcription Activator-Like Effector Nuclease
2. Engineered Meganucleases/Homing Endonucleases
3. CRISPR/Cas Nuclease

Figure 2. Genome-editing technologies. (Gaj, 2016)

Strategies for Gene Delivery

For gene therapy, it is far from enough to obtain therapeutic genes that can be stably expressed through gene editing. It is also important to successfully deliver the therapeutic genes to host cells, either integration into the genome or independent existence, and stably replicate and express. This requires direct inoculation, or by physical or chemical methods, or the most commonly used by viral vector-based gene delivery, which is designed to help therapeutic genes enter into the desired host cells.

Gene Therapy in Clinical Trials

The gene therapy industry is striving more than ever to define a path to the clinic and the market. Up to now, approximately 20 gene therapy products have been approved and almost 2600 human gene therapy clinical trials have already been reported around the world. These advancements raise great hope to treat devastating rare and metabolic disorders as well as incurable illnesses.

Genes and Associated Diseases

In cases where no other drug is effective, gene therapy may achieve long-lasting effects or even cure diseases with a one-time dose. As technology advances, the safety and efficacy of gene therapy are gradually improving. In summary, gene therapy gives people born with a genetic disorder or cancer the chance to live a normal life. So far, a series of genes and their related diseases have been discovered and studied, which provide a valuable theoretical basis for the development of gene therapy.

Gene Therapy for Human Genetic Disorders

Human genetic disorders, stemming from abnormalities in genes or chromosomes, significantly impact health, affecting physical, mental, and developmental traits. With over 10,000 known genetic disorders globally, comprising 1% of the population, these conditions pose a considerable public health challenge. Amidst the challenges, gene therapy emerges as an innovative medical frontier, aims to correct underlying defects, restore normal cellular functions, and enhance immunity, holding promise in treating or preventing genetic disorders.

Gene therapy by gene editing techniques has made great improvements in various diseases treatment in recent years. Scientists at Creative Biolabs lends every effort in our power to offer extensively high-quality gene editing services to global clients. For more detailed information, please directly contact us. We are more than happy to share our experience and expertise with our valued customers in related researches.

Reference

1. Gaj, T.; et al. (2016). Genome-Editing Technologies: Principles and Applications. Cold Spring Harbor Perspectives in Biology. 8(12): a023754.