The Process of Gene Augmentation Therapy?

The Process of Gene Augmentation Therapy?

There’s no new understanding of gene therapy. Actually, over 50 years of research and evolution have been undertaken by scientists and over 2,300 gene therapies clinical studies were currently planned, underway or completed.

Research in gene therapy, some of them very early, focuses in particular on several genetically-induced diseases, including disorders of blood clotting, e.g. haemophilia, cardiovascular disease, neurodegenerative disorders such as Parkinson’s disease, vision disorders and musculoskeletal disorders.

The Process of Gene Augmentation Therapy?

Millions of people with presently untreatable disorders may have hope because of the promise of gene therapy science.

What is a Gene Augmentation Therapy?

A procedure for correcting metabolic deficiencies caused by a missing or defective gene by having a healthy gene produce the necessary product without actually substituting that gene for the flawed or absent gene in the DNA.

Genetic Disease Understanding

It is important to know what a gene is until you can understand what gene therapy work is. There are trillions of cells in the human body. There is a nucleus containing chromosomes within a cell.

DNA, which is the body’s inheritance, is made of chromosomes. DNA segments are genes. Gene provides guidelines for protein building, controlling and sustaining the body.

There is a difference in the gene sequence of DNA occasionally. This is called a mutation which can lead to an incomplete or absent protein. A modification may be substituted, omitted or duplicated. Some mutations are inoffensive, but others may lead to genetic disease.

In short, gene therapy is a therapeutic method to cure or possibly avoid a genetic disorder.

Explore the gene therapy potential

One purpose of research into gene therapy is to find out whether a new or functional gene can be used to restore the function of a defective gene or to inactivate it One way of doing this is by bringing a gene into a cell.

A carrier, called a vector, is typically used for this. An altered virus will create a vector. It ensures that the viral genes are destroyed before the virus is used. It is possible to deliver vectors intravenously, that is, in a venous, by injecting them into a particular tissue in the body.

The six main vector types are:

  • Plasmid: Extrachromosomal circular DNA that is autonomous in the bacterial cell. Plasmids have a high number of copies, like pUC19, with 500-700 copies each cell.
  • Phage: Bacteriophage lambda linear DNA molecules. Without interrupting the life cycle it can be substituted with foreign DNA.
  • Cosmids: An additional circular extrachromosomal DNA molecule mixing plasmid and phage characteristics.
  • Bacterial Artificial Chromosomes. Based on bacterial mini-F plasmids.
  • Yeast Artificial Chromosomes. This is an artificial chromosome that contains telomeres (disposable buffers at the ends of chromosomes which are cut off during cell division) with origins of replication, a yeast centromere (part of a chromosome that links sister chromatids or a dyad), and a selectable marker for identification in yeast cells.
  • Human Artificial Chromosome. This type of vector is potentially useful for gene delivery into human cells, and a tool for expression studies and determining human chromosome function. It can carry a very large DNA fragment.

All engineered vectors come from a replication source (a replicator), a cloning site (which does not interrupt the replication or inactivation of critical markers by introducing foreign DNA) and a selectable marker (typically a gene that is antibiotic-resistant).

The Process of Gene Augmentation Therapy?

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