What is Gene Therapy?

-Replacing or altering defective genes -Focus on curing diseases such as...

-Muscular Dystrophy -Hemophilia -Cystic Fibrosis

-Techniques still need to be refined in humans

How does it work?

There are two types of gene therapy Germ Line Gene Therapy - introducing
functional genes into sperm cells or eggs, effectively changing their genome. The change is inheritable in future generations.

Somatic Gene Therapy - Adding the

functional genes to the patients somatic cells. The change is not inheritable.

How are the functional genes A carrier for delivered? the new genetic material or
vector is inserted into the cell.
gene to the nucleus and copy it into a chromosome

The vector is designed to carry the new The main type of vector is a simple
virus.

 Different viruses transfer genetic

material differently, so they are hand picked for the application at hand.

The main types used are retroviruses and adenoviruses. Retroviruses use RNA to transcribe genetic information, so reverse transcription must take place to create DNA. An issue however is that they can insert the genetic information anywhere in the chromosomes. Adenoviruses use DNA, and instead of copying genetic information into the chromosomes, they leave an extra piece of DNA lying in the nucleus.

 Many other types of virus can be used

such as adeno-associated viruses, pox viruses, herpes viruses and more.

There are non-viral vectors

Naked DNA - intramuscular injection of the new gene via DNA plasmids. Has a lower yield of expression but is much safer Lipoplexes, similar to the naked DNA method, but the DNA is covered with a lipid shell to protect it from harm and ease delivery. Oligonucleotides - are synthetically created, and designed to stop the faulty gene from functioning

 Non-viral Vector methods are designed

to change all of the bad cells by adding new genes to replace the old ones, or by coding the cells to destroy the disease causing gene.

Viral Vector methods infect a few cells,

which alters the gene to be non-disease causing, but the viral genetics are left as well, so the cell will reproduce the virus and help spread the genetic information throughout the body.

The techniques are not perfect

We still have a long way to go before

we can truly utilize gene therapy to its full ability.

Viral methods can, in rare cases, cause

disease in patients, such as leukemia.

We need to more fully understand the

genes that cause these diseases to be able to replace or destroy them.