Gene therapy

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1. Gene Therapy 2. Uses: MONOGENEIC DISORDERS AND COMPLICATED DISORDERS ã Muscular dystrophy. ã Cardiovascular diseases. ã Block neurological disorders. ã…
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  • 1. Gene Therapy
  • 2. Uses: MONOGENEIC DISORDERS AND COMPLICATED DISORDERS • Muscular dystrophy. • Cardiovascular diseases. • Block neurological disorders. • Eliminate infectious pathogens. Gene therapy is an experimental technique used in treatment of malfunctioning genes, whereby a deficient or defective gene is replaced by a working gene.
  • 3. Source: https://en.wikipedia.org/wiki/Gene_therapy
  • 4. Germ line Gene Therapy Altering the genetic makeup of a gene of either an egg or a sperm cell before fertilization, or altering the genetic composition of a blastomere during an early stage of its division. Advantages Disadvantages cure is inherited Error during the gene 'transplant'
  • 5. Source: http://genmed.yolasite.com/
  • 6. Somatic Gene Therapy Altering the genetic code or chromosomes of a person's somatic cells. Advantages less controversial Disadvantages use of viral vectors, is difficult.
  • 7. GENE DELIVERY • Ex-vivo Adeno-virus or lenti. • In-vivo lenti-virus. (non-div)
  • 8. Source: http://genmed.yolasite.com/
  • 9. VECTORS USED IN GENE THERAPY • RETRO VIRUS. • ADENOVIRUS. • ADENO-ASSOCIATED VIRUSES [AAVS]. • HERPES SIMPLEX VIRUS [HSV]. • ALPHAVIRUSES. • VACCINA OR POX VIRUSES. Source: http://geneticmutationsgenet herapy.yolasite.com/
  • 10. source: http://www.nature.com/
  • 11. Retroviruses • First virus used as vector. • Reverse transcription. • Genome copies integrated into host by integrase enzyme. • Zinc finger nuclease. • Gene therapy for SCID is vey much useful.
  • 12. ADENOVIRUS • with double stranded DNA genome that can cause respiratory, intestinal and eye infection. • Don’t incorporated into host cell genetic makeup. • Affect broader variety of cells. • Attacked by patient immune system, required high quantity. • Used for treating cancer of liver and ovaries.
  • 13. ADENO-ASSOCIATED VIRUSES [AAVS] • Small, icosahedral, single-stranded DNA adeno-associated virus. • Wild-type AAV contains a 4.7-kb. • 100 different capsids. • AAVs are small viruses from the Parvovirus family with a genome of single stranded DNA.
  • 14. HERPES SIMPLEX VIRUS [HSV] • Human neurotropic virus. • HSV makes an ideal vector as it can infect a wide range of tissues including muscle, liver, pancreas, and nerve and lung cells. • Able to infect neurons which are not rejected by immune system.
  • 15. Gene therapy vectors • ALPHAVIRUSES • Pox virus • NON-VIRAL OPTIONS • Particles carrying the therapeutic gene are injected intravenously and strong, high-gradient external magnets are used to capture the particles as they flow through the blood- stream.
  • 16. MAGNETIC NANOPARTICLE-BASED GENE DELIVERY Source: http://www.nature.com/
  • 17. GENETIC DEFECTS Source: http://images.wisegeek.com/
  • 18. Source: http://japha.org/
  • 19. Source: http://www.s-cool.co.uk/
  • 20. Source: http://www.dailymail.co.uk/ Gene therapy for damage retinal cells
  • 21. GENE THERAPY RISKS • The gene therapy took triggered a chain reaction in his immune system, resulting in hepatic and respiratory failure, and consequently, his death four days after being treated. • The genetically modified somatic cells decreased in amount. Because they are already differentiated and possess only a limited capability to multiply.
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