Somatic and germline gene therapy, Biology

Assignment Help:

All the cells present in the body contain genes and an approach targeted to modulate the expression of genes is known as gene therapy. This novel approach has emerged by the end of 20th century and comes up with a promise of treating a large number of genetic disorders by ultimately modifying the gene expression (Jin et al, 2008). The two forms of gene therapy are somatic and germ line.

Somatic gene therapy: During this therapeutic approach, the genetic diseases are treated by inserting recombinant genes into non-reproductive or somatic cells. This kind of approach is limited to the patient and does not get transmitted to the future generations of. A normal copy of the malfunctioning gene is inserted into the mutant cells due to which an improvement in the disease phenotype is observed (Dorin, 1996). In the recent past, this technique has been found to be successful in case of familial hypercholesterolaemia and adenosine deaminase deficiency. For treating these disorders hepatocytes and T cells have been used for gene transfer respectively (Dorin, 1996). It has also shown significant success in case of cystic fibrosis and cancer. Various vehicles, like viral and non viral vectors help in the introduction of the genetic material into the mutated cells (Elias and Annas, N.D). This therapeutic approach is categorized into three sections:

  • ex vivo - The genetic engineering of the cell is done outside the body and finally the cells are returned to the body. (Ex: blood cells)
  • in situ - The cells of the affected tissues are directly transfected with vectors carrying genetic material. (Ex. Tissues of the respiratory tract)
  • in vivo - The vector is directly injected into the blood stream and this method is still in the developmental stages (Anderson, N.D).

However this approach produces short term results as the tissues die and get replaced with other cells.


Related Discussions:- Somatic and germline gene therapy

Can you explain toxic alkaloids, Q. Can you explain Toxic alkaloids? Al...

Q. Can you explain Toxic alkaloids? Alkaloids are the nitrogen heterocycles which occur mainly m plants as their salts of common carboxylic aciak such as citric, lac

Explain about the terms - bursae and captacula, Explain about the terms - b...

Explain about the terms - bursae and captacula? Paired water-filled sac located at base of each arm of a brittle star. Cilia in bursae circulate water. Its surface is significa

Define essentiality of nitrogen for growth of micro-organism, Define Essent...

Define Essentiality of Nitrogen for Growth of Micro-Organism? Nitrogen is an essential atom in certain cellular macromolecules, e.g. amino acids, purines, pyrimidines, enzymes,

Fungi, what is primitive fungi?

what is primitive fungi?

Respiratory distress syndrome, Respiratory Distress Syndrome: Respirat...

Respiratory Distress Syndrome: Respiratory distress in a  newborn is  a challenging problem.  It  accounts for significant morbidity and mortality. It occurs  in  4 to 6 per c

Explain the working of specific acquired immunity, Working of Specific Acqu...

Working of Specific Acquired Immunity Primary lymphoid organs, as you already know, are thymus and bone marrow. Secondary organs are lymph nodes, spleen and lymphoid tissue. Wh

Explain about posterior wall, Explain about Posterior wall Posterior w...

Explain about Posterior wall Posterior wall separates the antrum from the infra-temporal fossa and contains two important structures. Posterior superior alveolar nerve and

Explain gametophyte, "In these autotrophs, sporophyte is the dominant gener...

"In these autotrophs, sporophyte is the dominant generation. Gametophyte is also photosynthetic and not dependent on sporophyte for nutrition."  These autotrophs are:  a.  P

Deciduous senescence - senescence, Deciduous Senescence - Senescence O...

Deciduous Senescence - Senescence Only the leaves senesce as in many trees. The senescence of leaves, or abscission occurs when at the base of a leaf a layer of cells is laid

Explain the human body system and organs, Body systems and organs and their...

Body systems and organs and their functions The human body is a large structural unit made up of many smaller parts. There are four kinds of smaller units - cells, tissues, org

Write Your Message!

Captcha
Free Assignment Quote

Assured A++ Grade

Get guaranteed satisfaction & time on delivery in every assignment order you paid with us! We ensure premium quality solution document along with free turntin report!

All rights reserved! Copyrights ©2019-2020 ExpertsMind IT Educational Pvt Ltd