Genes as Medicine Interactive Video

What was the first sign that Laura and Ryan Troxel noticed about their daughter Molly's health condition?

• She had difficulty hearing.

• 
  She had trouble walking.

• 
  She would look towards bright light.

• She was not responding to her name.

What is the cause of Molly's blindness according to the narrator?

• A lack of vitamins during pregnancy.

• 
  A mutation in the genetic instructions for a protein in her eyes.

• 
  An unknown virus affecting her vision.

• 
  A physical injury to her eyes.

According to Bennett, what is the big idea of gene therapy?

• To treat a disease by altering the physical symptoms.

• 
  To prevent the disease from spreading to other individuals.

• 
  To provide temporary relief from genetic disorders.

• To treat a disease at its root by altering the instructions that the cell is given.

What is the retina?

• 
  A muscle that controls the size of the pupil

• A part of the brain responsible for processing visual information

• 
  A light-sensitive layer of neural tissue at the back of the eye

• The clear front surface of the eye that covers the iris and the pupil

What is the role of viruses in the gene therapy described?

• They are used to strengthen the immune system against eye diseases

• They are used to deliver DNA or RNA across cell membranes

• 
  They are used to replace the entire DNA of the cell

• 
  They are used to improve the vision by altering the retina

What is Leber amaurosis?

• A type of gene therapy

• 
  A method for injecting genes

• A genetic condition causing child-onset blindness

• A breed of dogs

What causes Leber amaurosis in both humans and dogs?

• 
  A virus

• 
  Exposure to sunlight

• 
  A mutation in the RPE65 gene

• 
  Aging

How did Molly Troxel inherit the disease-causing mutation?

• From a virus

• 
  From a random genetic mutation after birth

• 
  

  From environmental factors

• 
  From both of her parents, each having one working copy and one mutated copy of the RPE65 gene

What unfortunate event occurred during a gene therapy trial at the University of Pennsylvania in 1999?

• The patient's immune system did not react to the viral invasion

• Jesse Gelsinger died due to an immune reaction to the viral vector

• The researchers decided to stop the trial voluntarily

• The therapy was unsuccessful.

What was the reaction of the field of gene therapy after the death of Jesse Gelsinger?

• Increased funding and support

• No change in the level of interest and support

• A halt in gene therapy research due to a loss of interest and funding

• A shift in focus to other types of therapy