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

Explain the nutritional and functional role of sulphur, Minerals :-  Sulfur...

Minerals :-  Sulfur Food Source       Widely distributed  Nutritional Functional role Essential nutrient: A constituent of the essential amino acids methionine and

Surface water sources and charactersitics, Most of the surface water origin...

Most of the surface water originates directly from precipitation in the form of rainfall or snow. Ground water from springs also contributes to the flow of most of the streams. The

Find initial osmotic pressure on room temperature of a cell, Find the initi...

Find the initial osmotic pressure at room temperature of a cell if the only ions present are CaCl2 on either side of the membrane. Assume the concentrastions for K+ and Cl- from Ta

Explain why this drug would eventualy kill a person, Dinitrophenol, DNP, wa...

Dinitrophenol, DNP, was prescribed in low doses in the 1940's to help obese patients lose weight. It's use for this purpose was discontinued when several users died. DNP makes the

Define starches, Normal 0 false false false EN-IN X-N...

Normal 0 false false false EN-IN X-NONE X-NONE MicrosoftInternetExplorer4

Amnion adaptation to terrestrial life, Q. Why can the amnion also be consid...

Q. Why can the amnion also be considered an adaptation to terrestrial life? The amnion is as well an adaptation to dry land since one of its functions is to prevent desiccation

Therapeutics for infective endocarditis, Therapeutic   Accurate identif...

Therapeutic   Accurate identification of the infecting organism is the key to successful treatment.  Antibiotics as per the sensitivity study in high doses IV is given.

Mineral nutrition, Normal 0 false false false EN-IN X...

Normal 0 false false false EN-IN X-NONE X-NONE

Social determinants of health - social exclusion, Social Determinants of He...

Social Determinants of Health - Social Exclusion Given that absolute poverty is a major determinant of ill-health, the resultant social exclusion is ‘psychologically damaging,

Biomolecules, How do you explain in the absence of aldehyde group in the pe...

How do you explain in the absence of aldehyde group in the pentaacetate of D-glucose? Ans) when you react glucose with acetic anhydrate it forms pentaacetate with 5 OH grops it tel

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