HHMI Genes as Medicine video assignment

What condition was diagnosed in Molly Troxel when she was a few months old?

• A heart condition

• A form of blindness

• A muscle disorder

• A respiratory issue

What is the primary goal of gene therapy as described in the video?

• To enhance physical appearance

• To treat diseases at their root

• To prevent all inherited diseases

• To replace damaged organs

Who was the first to realize the potential for gene therapy to restore vision in blind animals?

• James Barrat

• Jean Bennett

• Ryan Troxel

• Albert Maguire

What genetic mutation is associated with Molly's blindness?

• RPE65

• BRCA1

• TP53

• CFTR

Tags

NGSS.HS-LS3-2

What type of animal was used to test the gene therapy before human trials?

• Cats

• Mice

• Briard shepherds

• Rabbits

What tragic event occurred in 1999 that affected the gene therapy field?

• A successful human trial

• A breakthrough in gene therapy

• A patient died during a trial

• A new gene was discovered

How did Molly Troxel feel about the gene therapy procedure?

• Completely indifferent

• Excited and scared

• Reluctant and unhappy

• Confused and uncertain

What did Molly see after her procedure that was significant for her?

• The sun

• The moon

• Her parents' faces

• A television screen

How many patients have participated in RPE65 clinical trials with excellent results?

• Over 100

• About 40

• 25

• 50

What was the outcome of the RPE65 gene therapy as recommended by the FDA?

• It was rejected altogether

• It was approved for treatment

• It is still under review

• It was only approved for animals