The Very Basics Of Gene Therapy

Understanding The Basics Of Gene Therapy

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.

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.

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.

Types Of Gene Therapy

Somatic Cell Gene Therapy

Germ Line Gene Therapy

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.

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.

Diseases Which Can Be Treated

• Colour blindness.• Cancer.

• Haemophilia.• Thalassemia.

• Parkinson’s disease.• SCID.

• HIV and more.

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Presented To You By

Tyler Van Buren