How might we cure diseases in the future?

Using what we know about genes

• Pharmacogenetics/ Pharmacogenomics

• Gene Therapy

• Regenerative medicine

Pharmacogenomics, Pharmacogenetics

PERSONALIZED MEDICINEThis 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

makeup.

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 effects.

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 one.

Example

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 recombination.

The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function.

The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.

Regenerative Medicine STEM CELL THERAPY

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.

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 cells.

Regenerative medicine is already being used.

• Bone marrow transplants• ??????

So why can’t it be used to replace all faulty cells?

Stem cell controversy