The Very Basics Of Gene Therapy
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Transcript of The Very Basics Of Gene Therapy
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Understanding The Basics Of Gene Therapy
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Correcting Defective Genes• Replacing the defective
gene with a normal copy of it.
• Knocking out the gene or silencing its function.
• Inserting a new gene to prevent the disease.
• Repairing of the defective gene via selective reverse
mutation.
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Gene Therapy In The Early Days
• Insertion of human genes into bacteria.
• Transcription and translation of the gene into protein by
bacteria.• Insertion of the protein into
human cells.
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Modern Day Gene Therapy•Vector delivery of therapeutic gene into target human cell.•Infection of target cell with viral vector.•Insertion of vector gene into target cell.•Transcription and translation of functional proteins.
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Types Of Gene Therapy
Somatic Cell Gene Therapy
Germ Line Gene Therapy
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Somatic Cell Gene Therapy
•In-vivo (cells modified inside the body) or ex-vivo (cells modified outside the body).•Somatic cells are used and hence, results are not inherited by future generations.•Also, the results are short-lived as the cells are non-reproductive.
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Germ Line Gene Therapy
•Genes are sourced from germ line cells and hence, results are permanent.•Cells from a man’s sperm are injected with healthy gene.•These cells are put in a mouse, where they mature.•The healthy sperm cells are tested and used to fertilise women’s eggs.•Fertilised eggs inserted into a woman’s womb.
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Diseases Which Can Be Treated
• Colour blindness.• Cancer.
• Haemophilia.• Thalassemia.
• Parkinson’s disease.• SCID.
• HIV and more.
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Thank You For Watching
Presented To You By
Tyler Van Buren