PDF 20230926 132409 0000

Is altering genetic material
against the god's will ?

Medical science has detected many human diseases related to defective genes. These types of diseases are not
curable by traditional methods like taking readily available medicines. Gene therapy is a potential method to
either treat or cure genetic-related human illnesses
In 2015, researchers from Harvard and Boston Children's Hospital used gene therapy to restore basic
hearing in genetically deaf mice. They also improved the hearing level to 25 decibels, similar to a
whisper, using a unique vector called "Anch".
Gene therapy, originating in 1971, uses foreign DNA to target and treat diseases in a patient's tissue. It's a
promising but challenging field, largely driven by progress in recombinant DNA technology.
The Basic Process
There are several approaches to gene therapy. These are the following (Fliesler, 2017)
replacement of mutated gene that causes disease with a healthy copy of the gene
Inactivation of a mutated gone that is functioning improperly
Introducing a new gene into the body to help fight a disease
In general, a gene cannot be directly inserted into a human gene or cell. A gene is inserted into another gene
using a carrier or vector. At present, the most common type of vectors are viruses that have been genetically
changed to carry normal human DNA. Viruses have evolved a way of encapsulating and transporting their genes to
human cells in a pathogenic manner (Science Daily, 2017).
Two Types of Gene Therapy
There are essentially two forms of gene therapy. One of which is called somatic gene therapy. Somatic
gene therapy involves the manipulation of genes in cells that will be helpful to the patient but not
inherited to the next generation (Nimserger, 1988).
The other form of gene therapy is called germ-line gene therapy which involves the genetic
modification of germ cells or the origin cells that will pass the change on to the next generation (Your
Genome, 2017).
Somatic Gene Therapy
- For treatment of cystic Fibrosis, Muscular Dystrophy, Cancer & Certain infections diseases
Types:
1. Ex vivo ( in vitro)
2. In vivo
Ex-Vivo Gene Therapy
- Manipulation outside the body
- target specific organ/ tissue
For: 1. BM STEM CELLS 6. Corneal disease
2. Liver cells 7. Stroke
3. blood vessel smooth muscle cells 8. Multiple sclerosis
4. Tumor infiltrating lymphocytes 9. Treatment of leukemia
5. Blindness 10. Glaucoma
Principle: - Defective gene taken out of the body
- Therapeutic gene carried by the vector
- Transplanted back to the host
Procedure:
1. Isolation of defective gene
2. Therapeutic gene is inserted to defective gene through vector
3. Allow to grow
4. Transformed cell is injected back to the host
5. Genetically altered cell produce the desired proteins encoded by therapeutic gene
Advantages: 1. Target specific organ

2. Manipulated cell is immunocompatible
Disadvantage: 1. More invasive procedure

In-Vivo Gene Therapy
Applied to: 1. Liver 4. Lungs 7. Blood cells
2. Muscles 5. Spleen
3. Skin 6. Brain
Corrected Genes Delivered through:

1. Viral vector
2. Non- vector ( Physical Methods )
Advantage: Minimal invasion
- single step of injection of vector into desired organ
Disadvantages: 1. Non-specific target cell infection

2. Possibility of causing of toxicity of illiciting immune response
Factors to consider:
1. Uptake of therapeutic gene carrier vector by target organ/tissue

- requires appropriate vector
2. Intracellular Deggradation
- should reach the target tissue w/o degradation
3. Expression ability of target tissue

- therapeutic gene should be expressed in target tissue
Germline gene therapy
- This strategy modifies gene in reproductive cells. Hence the gene will be passed on the next generation. This
would enable correction of certain growth and fertility problem and a potential for preventing inherited
diseases.
STEM CELL GENE THERAPY

- are mother cells that have the potential to become any type of cell in the body. Stem cell has also a
ability to self renew or multiply while maintaining the potential to develop into other types of cell.
Stem cell has a different source but they all have a capacity to develop into multiple types of cell.
Stem cells are derived from different source
1. Embryonic STEM CELL

- came from a four or five-day-old human embryo that is in the blastocyst phase of development.
2. Somatic stem cell

- cells that exist throughout the body after the embryonic develop. You can found this all in different
tissues such as brain, blood and etc. This cell can also divide indefinitely somatic stem cell has a
limited ability to differentiate based on their tissues of origin.
Thank
You

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Is altering genetic material

against the god's will ?

Advantages: 1. Target specific organ

Disadvantage: 1. More invasive procedure

Corrected Genes Delivered through:

Disadvantages: 1. Non-specific target cell infection

1. Uptake of therapeutic gene carrier vector by target organ/tissue

3. Expression ability of target tissue

STEM CELL GENE THERAPY

1. Embryonic STEM CELL

2. Somatic stem cell

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