Targeted Gene Therapy

Introduction

Many diseases are caused by the improper function of a single gene. Some examples include:

· In sickle cell anemia, misshapen blood cells carry less oxygen, which can cause pain and progressive organ damage. It’s caused by a mutation in the hemoglobin gene HBB.

· Thanks to 2014’s ice bucket challenge, scientists have uncovered the gene likely responsible for the progressive neuromuscular disorder, ALS: NEK1.

· A mutation in the gene CFTR results in Cystic Fibrosis, causing health problems because of abnormal mucus production in the lungs and digestive system.

For all of the above disease, gene therapy could hold the cure. To cure the disease, the correct gene can be selectively delivered to the cells (or tissues) to restore normal function. Such targeted gene delivery can be used as therapy for all the above diseases. Delivering the gene to the wrong cells or tissue would be ineffective or even cause harm to the patient.

Programming Challenge

The goal of this challenge is to develop a model for gene therapy. The robot will act as a model of a viral vector, delivering a therapeutic gene to a specific tissue.

Your robot needs to identify the therapeutic gene (pom pom), enter the "blood stream" (black line), and "deliver" the therapeutic gene to the correct tissue, signified by the color section. The color of each therapeutic gene and its target tissue match (see below):

Red HBB red blood cells

Yellow NEK1 motor neurons

Green CFTR epithelial cells of lungs and digestive track

Requirements:

The robot must identify which gene is presented inside the black box (it will be randomly selected by the instructor). The robot must stop or make a sound once it finds the target tissue of the same color to signal that it "delivered" the gene. The robot must use line following (using a color sensor to direct the robot to follow along a black line) to move along the black line as it simulates moving through the bloodstream.

SIM Background Download Here (png)

SIM Background Download Here (jpeg)

Challenge Mat Materials:

1 challenge mat

1 red, 1 green, 1 yellow pompom (randomly selelcted therapeutic gene)

Questions to guide your reading:

How can gene therapy be used to treat diseases?
What are some other applications of gene therapy other than curing defective genes?
Is gene therapy safe? What are the limitations of gene therapy?
How is gene therapy currently being used?