Biology 1202B Lecture Notes - Lecture 21: Hematopoietic Stem Cell Transplantation, Cep290, Genome Editing

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2 Apr 2019
Department
Professor
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?
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