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

Systems theory - accident causation, Systems Theory - Accident Causation ...

Systems Theory - Accident Causation The systems theory established extremely strong relationship between system and accident. A properly functioning system may avoid accidents

What are autotrophic beings? what are heterotrophic beings, What are autotr...

What are autotrophic beings? What are heterotrophic beings? The Autotrophic beings are those that can produce their own food that is that make organic material from inorganic c

Central carbons participate in the peptide bond, Q. Do the -H groups bound ...

Q. Do the -H groups bound to the central carbons participate in the peptide bond? The central carbons themselves, the -R groups and the hydrogen attached to the central carbons

Describe the maturity stages in fish, Question 1: (a) Describe the mat...

Question 1: (a) Describe the maturity stages in fish. (b) "k" and "r" selection strategies are commonly used to describe the response of fish to biotic and abiotic cond

Define the elimination of dna ligase activity in a cell, Which of the follo...

Which of the following results from the elimination of DNA ligase activity in a cell? A. The oligonucleotide primers will not be removed from the genome - as they often contain

Solid fuels, Primary and secondary solid fuels are widely used in domestic ...

Primary and secondary solid fuels are widely used in domestic and industrial purpose. Wood and coal constitute the important primary solid fuels. The important secondary solid fuel

What is the primary structure of a protein, What is the primary structure o...

What is the primary structure of a protein? What is the significance of the primary structure? The primary protein structure is the linear sequence of amino acids that form the

Explain the uses of isp in seafood products, Explain the Uses of ISO in Sea...

Explain the Uses of ISO in Seafood products  Seafood products  The most significant application in this category is the use of ISP in fish sausage and surimi based restructu

What is the approximate number of amino acids, What would be the approximat...

What would be the approximate number of amino acids of a protein with a mass of 203 kd? Assume an average molecular weight for amino acids of 110 grams/mol.

Determine the preservation changes while cooking, Determine the Preservatio...

Determine the Preservation Changes While Cooking? While cooking, two preservation changes (at least) occur which include: 1.  Destruction or reduction of microorganisms 2

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