Biology 1202B Lecture Notes - Lecture 21: Hematopoietic Stem Cell Transplantation, Cep290, Genome Editing
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2 Apr 2019
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Mon, Mar. 25/19 – Lecture 21: DNA Technologies I
• DNA technologies does not mean that a new technology can solve all of the problems it just means
we have another means for a potential cure or another way that does not work
• Knowing the sequence of genes, and up to date technology is not sufficient for finding cures
Gene Editing, Dying Girl
• Very aggressive form of leukemia, unsuccessfully through chemotherapy
• Palliative care: Nature care for death
• Talen and Endonucleases were used
CRISPR
• Immune system of bacteria
• Cuts a piece of DNA and can either replace it or remove it
• Very easy, efficient, cost effective, precise
• Girl dying of a severe form of leukemia
o Gene editing
▪ Edited genome of Donor T cells --> Designer T cells
▪ Designer T cells attacked her tumor
▪ Immune systems sees the cancer cells as itself so it does not attack it
▪ Therefore if we can make the cancer cells alien to the immune system we can kill off the
cancer
▪ She was on a drug that was killing her own T Cells
▪ Highly specific designer T cells to the tumor and it became invisible to the drug but it could
see the cancer cell
▪ The Designer T Cells killed the cancer
▪ Took all her bone marrow out (stem cells) and re-administered bone marrow
transplant after the Designer T cells were put in through IV
Historic Milestones in Genomic Medicine
• Sequencing freedom, we can do pretty much anything
• CRISPR is used for congenital blindness
• Not just one gene but there are multiple factors (epigenetics, somatic mutations etc.)
o Different people have different factors
o LCA10 – CEP290
o Editing somatic cells is ethical
o Editing germline cells in unethical
▪ Have not mastered the CRISPR technique
▪ Do not know the side effects
▪ Medicinal (diseases) or changes for designer babies?
• RIch vs. Poor
• Gene pool manipulation
• Any editing leading to heritable changes is momentarily banned
• Biological weapon?
• Designer pathogens?