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Page 1: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

GENE THERAPY

Page 2: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

What is gene therapy?

• Gene therapy is the introduction of normal genes into cells that contain defective genes

Page 3: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

Why gene therapy?

• To supply the cells with the correct directions to make a missing protein product

Page 4: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

How?2 Delivery Systems

1. In vivo occurs within the organisms

Page 5: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

2. Ex vivo occurs outside the organism

Page 6: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

Who?The First Case

• September 14th, 1990– Ashanti DeSilva was treated for SCID

• Sever combined immunodeficiency • Gene is located on chromosome #22

– Doctors removed her white blood cells, inserted the missing gene into the WBC, and then put them back into her blood stream.

– This strengthened her immune system– Only worked for a few months

Would this be considered in vivo or ex vivo?

Page 7: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

Vectors

Vectors are carriers. (ex. insects are considered vectors for pollen)

Vectors transport the DNA into the body or into the cells.

What was the vector in the transformation lab?

Page 8: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

Viruses are frequently used as vectors because:

• They insert their viral DNA

into cells as a normal part of their lifecycle.

So, why not let the virus inject the good gene into a cell?

Imagine this lifecycle takingplace in the human body witha good gene. Explain whatwould happen?

Page 9: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

General concernsThe Food and Drug Administration (FDA) has not yet approved any human gene

therapy product for sale.

Major problems with gene therapy:

2) Immune response. It reduces gene therapy effectiveness and makes repetitive rounds of gene therapy useless

3) Viral vectors . Immune and inflammatory responses, also fears that viral vector may recover disease-causing ability

5) Target. One does not have control over where the gene will be inserted into the genome

1) Short-lived. Very hard to achieve any long-term benefits

4) Multigene disorders. Disorders such as heart disease, Alzheimer's disease, arthritis, and diabetes, are caused by the combined effects of many genes.

Page 10: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

Now!!

More than 5000 patients have been treated in last ~12 years worldwide

Page 11: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

Progress Slowed

• In 1999, 18-year-old Jesse Gelsinger died from multiple organ failure 4 days after treatment for an enzyme deficiency.– Death was triggered by severe

immune response to adenovirus vector

• January 2003, halt to using retrovirus vectors in blood stem cells because children developed leukemia-like condition after successful treatment for X-linked severe combined immunodeficiency disease (bubble boy disease)

Page 12: GENE THERAPY. What is gene therapy? Gene therapy is the introduction of normal genes into cells that contain defective genes

Progress Continues

• Cystic Fibrosis

• Alzheimers

• Parkinsons

• Sickle Cell

You will read more…….


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