Genes as Medicine Video Quizizz

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

• She had difficulty hearing.

• She would look away from light.

• She had trouble walking.

• She was not responding to her name.

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

• A physical injury to her eyes.

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

• An unknown virus affecting her vision.

• A lack of vitamins during pregnancy.

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

• To provide temporary relief from genetic disorders.

What is the retina?

• A muscle that controls the size of the pupil

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

• A part of the brain responsible for processing visual information

• 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 replace the entire DNA of the cell

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

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

What is Leber amaurosis?

• A type of gene therapy

• A genetic condition causing child-onset blindness

• A method for injecting genes

• A breed of dogs

What causes Leber amaurosis in both humans and dogs?

• A virus

• A mutation in the RPE65 gene

• Exposure to sunlight

• Aging

How did Molly Troxel inherit the disease-causing mutation?

• From a virus

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

• From a random genetic mutation after birth

• From environmental factors

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

• A) The therapy was a complete success

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

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

• D) The researchers decided to stop the trial voluntarily

What was the outcome of the gene therapy experiments on Briard Shepherds with mutations in the RPE65 gene?

• A) The dogs were able to navigate simple environments

• B) The dogs showed no improvement

• C) The dogs' condition worsened

• D) The dogs developed a different health issue