H2O.ai CEO/Founder: Sri Ambati Keynote at Wells Fargo Day
Lesson 11 Gene Therapy
1. Gene Therapy. To understand how gene therapy may make it possible to treat certain genetic disorders
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3. Chromosome 7 CF Allele Find the CF Allele on a human chromosome. Use chromosomes from someone without CF. Cut out the normal alleles. Make lots of copies Put these copies into cells of the CF patients - (Gene Modification)
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7. Gene therapy – hope for the future? Gene therapy could be soon be the magic cure for people with genetic diseases. It works by putting the corrected genes they need into the person’s cells. Gene therapy has already cured a few babies of the genetic disease SCID. Unfortunately two babies who had the treatment in France have developed serious blood diseases. This could be because of their gene therapy. Some doctors think that the new genes could affect the cells in a way we don’t yet understand. So both France and the USA have stopped their trials of gene therapy for SCID. A mother whose son was waiting for this treatment in France is very upset. ‘It was probably nothing to do with the treatment. It’s just a coincidence. There is no suitable bone marrow transplant for my baby. It’s not fair that he can’t try gene therapy,’ she said.
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