MCELLBI C44 Lecture Notes - Lecture 18: Hematopoietic Stem Cell Transplantation, Wild Type, Gene Therapy
21 Mar 2017
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Document Summary
Pull out bone marrow, stick in a virus. One paediatric patient in france has been cured of sickle cell disease > major step forward. If you put in dna randomly, it doesn"t always work. (like throwing the 5th tyre at a car with flat tyre and hoping it works) The genome is huge! (would take a century to read it one letter/second) Human genome is highly resistant to change. People with specific disease that make them children of the night : They are acutely sunlight sensitive (need 50"000spf if cloudy to avoid tumours) Our dna is continuously damaged by nature and the environment, but our cells have an entire molecular army to repair this damage! So bad, that nature has 2 separate repair mechanisms: Put the two ends back together (but loses or adds one or two base pairs by accident, that means there is a change in the human genome)
