Gene therapy

gene therapy

1. Gene Therapy

2. Uses: MONOGENEIC DISORDERS AND COMPLICATED DISORDERS Muscular dystrophy. C ardiovascular diseases. B lock neurological disorders. E liminate 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 A ltering 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://geneticmutationsgenetherapy.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] S mall , 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] H uman 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. A ble 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. T he genetically modified somatic cells decreased in amount. Because they are already differentiated and possess only a limited capability to multiply.