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How might we cure diseases in
the future?
Using what we know about genes
• Pharmacogenetics/ Pharmacogenomics
• Gene Therapy
• Regenerative medicine
Pharmacogenomics, Pharmacogenetics
This relatively new field
combines pharmacology
(the science of drugs)
and genomics (the study
of genes and their
functions) to develop
effective, safe
medications and doses
that will be tailored to a
person’s genetic
For example:
Hannah doesn’t feel well
So she goes to see Dr. Ramirez
Dr. Ramirez examines Hannah and decides that she
needs antibiotics to make her better
There are many types of antibiotics and in the past Dr.
Ramirez had to make an educated guess as to which
one would work best for Hannah and which one might
give her unwanted side effects.
Using pharmacogenomics/pharmacogenetics, Dr.
Ramirez could look at Hannah’s DNA to determine
which antibiotic would work best and not cause side
One size does not fit all
Can you think of a medicine that
doesn’t work for you?
Gene Therapy
Gene therapy is the insertion of genes into an
individual's cells and tissues to treat a disease
and hereditary diseases in which a defective
mutant allele is replaced with a functional
A normal gene may be inserted into a
nonspecific location within the genome to
replace a nonfunctional gene. This approach
is most common.
• An abnormal gene could be swapped for a
normal gene through homologous
The abnormal gene could be repaired
through selective reverse mutation,
which returns the gene to its normal
The regulation (the degree to which a gene is
turned on or off) of a particular gene could be
Regenerative Medicine
Regenerative medicine focuses on the repair,
replacement or regeneration of cells, tissues,
or organs. It uses a combination of
approaches including soluble molecules, gene
therapy, stem cell transplantation, tissue
engineering, and the reprogramming of cell
and tissue types.
Human Embryonic Stem Cells Source: Public Library of Science
(via Wikimedia Commons) under a Creative Commons Attribution
2.5 license
Stem Cells
• embryonic stem cells, In a developing embryo,
stem cells can differentiate into all of the
specialized embryonic tissues
• adult stem cells, In adult organisms, stem cells
and progenitor cells act as a repair system for
the body, replenishing specialized cells.
• cord blood stem cells, which are found in the
umbilical cord and act similarly to adult stem
Human Embryonic Stem Cells Source: Public Library of Science
(via Wikimedia Commons) under a Creative Commons Attribution
2.5 license
Regenerative medicine is already
being used.
• Bone marrow transplants
• ??????
So why can’t it be used to replace all faulty cells?
Stem cell controversy