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Gene Therapy PowerPoint Presentation

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Description : Check out this medical PowerPoint presentation titled "GENE THERAPY".
This medical PowerPoint presentation talks about Gene therapy, a type of medical intervention that involves altering or replacing a persons genes to treat or prevent a disease. The goal of gene therapy is to introduce healthy genes into cells that have defective or missing genes, thereby correcting the underlying cause of a disease.
There are different types of gene therapy, but the most common approach involves using a carrier, such as a virus, to deliver the healthy genes into the patients cells. The carrier is modified to carry the correct version of the gene, and once it is injected into the patients body, it infects the target cells and delivers the therapeutic gene.
Gene therapy has the potential to treat a wide range of diseases, including genetic disorders, cancers, and viral infections. However, it is still an experimental field and many challenges need to be addressed, such as ensuring the safety and efficacy of the treatments, as well as developing delivery methods that can effectively target specific cells and tissues.
Despite these challenges, gene therapy has already shown promise in treating several diseases, such as severe combined immunodeficiency (SCID), a rare genetic disorder that causes a weakened immune system, and some types of inherited blindness. Ongoing research is exploring the potential of gene therapy to treat many other diseases, and it is hoped that it will eventually become a widely available treatment option.

Tags : Gene therapy, gene, Genetics, Genetics PowerPoint presentation

Published on : Mar 27, 2023 | Views : 1718 | Downloads : 4

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Slide 1-

GENE THERAPY
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In humans Cancer 69%
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General concerns Four major problems with gene therapy:
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Gene therapy could be very different for different diseases Gene transplantation (to patient with gene deletion) Gene correction (To revert specific mutation in the gene of interest) Gene augmentation (to enhance expression of gene of interest) Targeted killing of specific cells by introducing killer gene Gene ablation – targeted inhibition of gene expression
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Gene therapy In vivo Ex vivo
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in vivo and ex vivo schemes http://laxmi.nuc.ucla.edu:8237/M288/SChow_4_10/sld005.htm
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Transgenes Integrated Not integrated - stable expression; may provide a cure - expression is transient; repeated treatments necessary - random insertions in heterochomatin can be inactivated; In euchromatin -- Can disrupt important host genes; Long-term consequences are unknown for episomes (plasmids) random mutagenesis not an issue
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How episomes and integrated trasgenes behave in dividing cells Integral transgene Episome Loss of plasmid
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Influences on choice of vector high efficiency viral vectors for gene replacement therapy of monogenic diseases (cystic fibrosis; SCID; hemophilia…)
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Desirable characteristics of gene delivery vector 1. High titer or concentrations (>108 particles/ml) 3. Precise and stable introduction of transgene 2. Easy and reproducible method of production 4. Vector should not elicit immune response in the host
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Methods of gene delivery (therapeutic constructs) -- Injection of naked DNA into tumor by simple needle and syringe -- DNA transfer by liposomes (delivered by the intravascular, intratracheal, intraperitoneal or intracolonic routes) -- DNA coated on the surface of gold pellets which are air-propelled into the epidermis (gene-gun), mainly non applicable to cancer -- Biological vehicles (vectors) such as viruses and bacteria. Viruses are genetically engineered so as not to replicate once inside the host. They are currently the most efficient means of gene transfer.
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MOST COMMON VIRAL VECTORS Retroviruses Adenoviruses Adeno-associated viruses Herpes simplex viruses
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Retroviral vectors are able to infect dividing cells only Good for cancer gene therapy Nevertheless, retroviruses are most often used vectors for common disease gene therapy Every therapeutic construct should include safety features In dividing cells nuclear membranes are broken down, so viral genome can enter and integrate into the chromosome Preintegration complex of retroviruses non able to penetrate nuclear membrane. Infection of dividing cells only
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Drawbacks of using a pseudotyped retroviral vectors Host range now is too broad. Cell-specific targeting not possible, but we can use it for ex vivo approaches. 3. G protein of VSV is toxic for cell pseudotypes could be produced only by already dying packaging cells (overcome by inducible promotors) 2. G protein of VSV is very immunogenic (so, it’s one-time approach) Other pseudotypes are available: HFV – human foamy virus, HIV-1, LCMV (lymphocytic chiriomeningitis) – non toxic for cells
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Lentiviral vectors Lentiviruses are retroviruses that can infect both dividing and nondividing cells Preintegration complex of lentiviruses can get through the intact membrane of the nucleus of the target cell. Able to infect nondividing or terminally differentiated cells such as neurons, macrophages, hematopoietic stem cells, retinal photoreceptors, and muscle and liver cells Example of lentiviruses: HIV-1 (infects T-helper cells) – AIDS. Good feature – no immune response!
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ADENOVIRUSES non-enveloped viruses containing a linear double stranded DNA genome 40 serotypes known; most producing respiratory infections in humans subgroup C serotypes 2 or 5 are predominantly used as vectors can infect both dividing and nondividing cells 12 antenna-like fiber projections for virus attachment www.nobel.se
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Problems with adenoviral vectors 1. Cannot integrate with the host cell genome expression from adenoviral vectors is transient (5-10 days) due to immunoclearance of the virus in vivo hepatic gene delivery to hemophilia B dogs. Days posttreatment
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Adeno-associated virus (AAV) -- does not stimulate inflammation in the host -- does not elicit antibodies against itself -- can enter non-dividing cells -- integrates successfully into one spot in the genome of its host (on chromosome 19 in humans). Can be ideal as: How to make expression tissue specific?