From Group 2

Protein Synthesis
Gene
DNA RNA
coding
template

Protein

Two Major Application 1) Gene therapy 2) Stem cell

Gene Therapy
Also known as somatic gene therapy Using genes as medicine

Treating genetic disease where the faulty gene is fixed, replaced or

supplemented with a healthy gene

Uses of Gene Therapy

Replace missing or defective genes Deliver bacterial or viral genes as a form of vaccination Deliver genes that speed the destruction of cancer cell Supply genes that causes cancer cells to revert back to normal cells

How is gene therapy done?

Ex vivo technique
Involves surgical removing cells
Injecting or splicing the new DNA

Letting them divide in cultures

In vivo technique
Requires no surgery Therapeutic DNA is injected into body cells

Usually via one of two types of viruses

- retrovirus

- adenovirus

Aspect of Gene Therapy

1. Gene Splicing
A method cutting the DNA of a gene to add base pairs Restriction enzymes cut specific sequence of DNA DNA ligase join the new genetic sequence into the broken chain Important uses: production of insulin and growth hormone

2. Gene Silencing

Also called antisense technology

A method inactive gene that may cause disease or be defective

Make an RNA strand complementary to the mRNA

This synthesized RNA will attach to mRNA and prevent that portion of mRNA creating the gene on the duplicate DNA strand in reverse transcription Treat several viruses: Chicken Pox, Hepatitis and AIDS

Class of undifferentiated cells that are able to differentiate into specialized cell types. Two main sources :
- Embryonic stem cells - Adult stem cells

Capable of dividing and renewing themselves for long period Unspecialized Can give rise to specialized cell types

Adult stem cells are undifferentiated cells found throughout the body that divide to replenish dying cells and regenerate damaged tissues.

Embryonic stem cell are pluripotent stem cells

derived from the inner cell mass of the blastocyst,

an early stage embryo.

Human embryos reach the blastocyst stage 4-5 days post fertilization.

Which time they consists of 50-150 cells.

Isolating the embryoblast or inner cell mass

Transferred into plastic laboratory culture dish that

contains a nutrient broth (culture medium)

Cells divide and spread over the surface of the dish. Inner surface of the culture dish is typically coated with mouse embryonic skin cells that have been treated so they will not divide.

The mouse cells in the bottom of the culture dish provide the cells sticky surface for attachment. Removed gently and plated into several fresh culture dishes. This process is repeated.

Embryonic stem cells that have proliferated in cell

culture for a prolonged period of time without

differentiating (pluripotent)

Given their unique regenerative abilities, stem cells offer new potentials for treating diseases such as diabetes, and heart disease.

Human stem cells could also be used to test new drugs.

Generation of cells and tissues that could be used for cell-based therapies.