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  • Gene therapy may be used for treating, or even curing, genetic and acquired diseases like cancer and AIDS by using normal genes to supplement or replace defective genes or to bolster a normal function such as immunity.
  • It can be used to target somatic cells (i.e., those of the body) or gametes (i.e., egg and sperm) cells. In somatic gene therapy, the genome of the recipient is changed, but this change is not passed along to the next generation.
  • In contrast, in germline gene therapy, the egg and sperm cells of the parents are changed for the purpose of passing on the changes to their offspring.
  • There are basically two ways of implementing a gene therapy treatment:

Ex vivo, which means “outside the body” :

  • Cells from the patient’s blood or bone marrow are removed and grown in the laboratory. They are then exposed to a virus carrying the desired gene.
  • The virus enters the cells, and the desired gene becomes part of the DNA of the cells. The cells are allowed to grow in the laboratory before being returned to the patient by injection into a vein.

In vivo, which means “inside the body” :

  • No cells are removed from the patient’s body. Instead, vectors are used to deliver the desired gene to cells in the patient’s body. GENE THERAPY and PHARMACOGENOMICS


Gene delivery tools:

  • Genes are inserted into the body using gene carriers called vectors. The most common vectors now are viruses, which have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner.
  • Scientists manipulate the genome of the virus by removing the disease-causing genes and inserting the therapeutic genes.
  • However, while viruses are effective, they can introduce problems like toxicity, immune and inflammatory responses, and gene control and targeting issues.

High costs:

  • Since gene therapy is relatively new and at an experimental stage, it is an expensive treatment to undertake.

Limited knowledge of the functions of genes:

  • Scientists currently know the functions of only a few genes. Hence, gene therapy can address only some genes that cause a particular disease.
  • Worse, it is not known exactly whether genes have more than one function, which creates uncertainty as to whether replacing such genes is indeed desirable.

Multigene disorders and effect of environment:

  • Most genetic disorders involve more than one gene. Moreover, most diseases involve the interaction of several genes and the environment.
  • For example, many people with cancer not only inherit the disease gene for the disorder, but may have also failed to inherit specific tumor suppressor genes.
  • Diet, exercise, smoking and other environmental factors may have also contributed to their disease


  • Pharmacogenomics is the study of how the genetic inheritance of an individual affects his/her body’s response to drugs.
  • The vision of pharmacogenomics is to be able to design and produce drugs that are adapted to each person’s genetic makeup.


Development of tailor-made medicines:

  • Using pharmacogenomics, pharmaceutical companies can create drugs based on the proteins, enzymes and RNA molecules that are associated with specific genes and diseases.
  • These tailor-made drugs promise not only to maximize therapeutic effects but also to decrease damage to nearby healthy cells. GENE THERAPY and PHARMACOGENOMICS

More accurate methods of determining appropriate drug dosages:

  • Knowing a patient’s genetics will enable doctors to determine how well his/ her body can process and metabolize a medicine.
  • This will maximize the value of the medicine and decrease the likelihood of overdose.

Improvements in the drug discovery and approval process:

  • The discovery of potential therapies will be made easier using genome targets. Genes have been associated with numerous diseases and disorders.
  • With modern biotechnology, these genes can be used as targets for the development of effective new therapies, which could significantly shorten the drug discovery process.

Better vaccines:

  • Safer vaccines can be designed and produced by organisms transformed by means of genetic engineering.
  • These vaccines will elicit the immune response without the attendant risks of infection. They will be inexpensive, stable, easy to store, and capable of being engineered to carry several strains of pathogen at once.



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