gene therapy

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Gene therapy :



AS SAID BY JAMES WATSON “We used to think that our fate was in our stars, but now we know that, in large measure, our fate is in our genes”

Genes :

Genes Unit of heredity in a living organism Normally resides on a stretch of DNA that codes for a type of protein or for an RNA chain Hold the information to build and maintain an organism's cells and pass genetic traits to offspring A locatable region of genomic sequence When altered causes dysfunction of a protein

Gene therapy :

Gene therapy Insertion of normal DNA directly into cells to correct a genetic defect Several approaches are used for treeating faulty genes A normal gene may be inserted into a nonspecific location within the genome An abnormal gene could be swapped for a normal gene The abnormal gene could be repaired through selective reverse mutation The regulation of a particular gene could be altered.


HISTORY 1970’s 1983 September 14, 1990 1990 Gene surgery Lesch-Nyhan disease First gene therapy Melanoma

Basic Process of Gene Therapy :

Basic Process of Gene Therapy Several approaches to gene therapy are being tested Replacing a mutated gene that causes disease with a healthy copy of the gene Inactivating, or “knocking out,” a mutated gene that is functioning improperly Introducing a new gene into the body to help fight a disease




STEPS OF GENE THERAPY A vector delivers the therapeutic gene into the patient’s target cell. The target cells become infected with the viral vector The vector’s genetic material is inserted into the target cell Functional proteins are created from the therapeutic gene causing the cell to return to a normal state

Steps of gene therapy:

Steps of gene therapy

  Types of gene therapy :

Types of gene therapy Somatic gene therapy Germline gene therapy Stem Cell research

Vectors :

Vectors Plasmids used in genetic engineering are called vectors Plasmids serve as important tools in genetics and biotechnology labs Vectors used for gene therapy are Viral Non.viral

Viral Vectors:

Viral Vectors All viruses attack their hosts and introduce their genetic material into the host cell machinery The host cell will carry out these instructions and produce additional copies of the virus Remove the genes in the virus that cause disease Numerous problems and risks exist that prevent gene therapy using viral vectors Replace those genes with genes encoding the desired effect

Viruses used for gene therapy:

Viruses used for gene therapy Retroviruses Adenoviruses Adeno-associated viruses Herpes simplex viruses Retrovirus Adenovirs Herpes simplex virus

Non-viral vectors :

Non-viral vectors Non-viral methods present certain advantages over viral methods Low levels of transfection and expression of the gene held non-viral methods at a disadvantage Following types are used for non-viral vectors Naked DNA Oligonucleotides Lipoplexes and polyplexes

Naked DNA :

Naked DNA Simplest method of non-viral transfection Intramuscular injection of a naked DNA plasmid Expression has been very low in comparison to other methods of transfection

Oligonucleotides :

Oligonucleotides Use of synthetic oligonucleotides in gene therapy is to inactivate the genes involved in the disease process The inactivation of gene can be done either by disrupting the transcription process or by disrupting the translation process

Lipoplexes and polyplexes :

Lipoplexes and polyplexes They have the property of protecting DNA from undesirable degradation during the transfection process Most common use of lipoplexes has been in gene transfer into cancer cells

Advantages of Gene Therapy :

Advantages of Gene Therapy Helps in the treatment of genetic diseases In Treatment of cancer

Disadvantages of Gene Therapy :

Disadvantages of Gene Therapy Genetic testing is producing much controversy and raising many ethical and legal problems People are opposed to gene therapy because they find it as something foreign and scary

Genetic Defects that are Candidates for Gene Therapy :

Genetic Defects that are Candidates for Gene Therapy

  What are some recent developments in gene therapy research? :

What are some recent developments in gene therapy research? Nanotechnology + gene therapy yields treatment to torpedo cancer. March, 2009 Results of world's first gene therapy for inherited blindness show sight improvement. 28 April 2008 Reengineering of immune cells, called lymphocytes, to target and attack cancer cells New gene therapy approach repairs errors in messenger RNA derived from defective genes C reation of tiny liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane

References :

References Burdette, Walter J. The Basis for Gene Therapy. Springfield: Charles C Thomas, 2001. Crayton, Stephanie. “First Clinical Trial Of Gene Therapy McCormack, Matthew P. “Activation of the T-Cell Oncogene Peel, David. “Virus Vectors & Gene Therapy



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