Genes as Medicine | HHMI BioInteractive video

Watch the story of how gene therapy restored the sight of a nearly-blind young patient. Told from the perspective of two researchers who spent over 25 years working to develop this breakthrough technology, this short film chronicles their successes and challenges, and illustrates how the method works to treat inherited conditions.

Gene therapy is a method for treating inherited diseases by delivering corrective versions of genes to patients. The development of this technology has been on a roller coaster of advances and setbacks over the last two decades. Few researchers are more familiar with that roller coaster than Dr. Jean Bennett and Dr. Albert Maguire at the University of Pennsylvania. They have focused their careers on developing a successful gene therapy for an inherited form of childhood blindness called Leber Congenital Amaurosis (LCA). On October 12, 2017, science advisors to the U.S. Food and Drug Administration (FDA) unanimously agreed that the benefits of this gene therapy far outweigh the possible risks. If approved by the FDA, the LCA treatment will become the first gene therapy available to treat an inherited disease.

This film tells the story of how the LCA gene therapy was developed. Students will learn how autosomal recessive conditions are inherited, how scientists can use modified viruses to deliver human genes to cells, what makes the eye an ideal tissue for gene therapy, and how model organisms are used to test treatments before they are tested in patients.

[KailashBP]

"I was hoping for perfection ... but what I have now is perfect" Molly

Lovely.

[kreiHibiscus]

i think the only people who actually watch this is for school purposes










at least according to the comments

[kyledeleon]

I find it ironic that her name is Dr. JEAN

[pramodsingh]

Genes as Medicine - Please god make this more and more successful...Dr Jean so nice discovery..Let this be used for human in need.

[NewMessage]

Just when watching the news seems to have completely drained me of any hope for humanity, I pop on YouTube to find this, and I'm reminded that there's still a massive reservoir of hope out there.

[themodernman]

IF this was your cyber day assignment, thumbs up.

[kaylahouston]

I think it is absolutely amazing how science has changed even over the last few years. Finding a cure and being able to edit a genetic mutation is unbelievable and so needed to help people with these gene mutations. Specifically, because humans are so complex and I especially thought it was interesting that there are so many different types of jeans and doctor/scientist that specialize in just the retina of your eye.

[freddiesavage]

This gives me so much hope for the future of gene therapy. If we can help our own bodies to produce better people then we can have more productive lives.

[khamis]

anyone else watching this for google classroom

[jackstilwell]

That is one of the sweetest, yet simultaneously amazing things I think I've ever seen

[Goldfox]

for class time stamps 4:30 6:10 7:30 14:00

[isabellamahdi]

I thought that this was a very interesting video. I especially liked how it was a true story and how it demonstrated how gene therapy works.

[stargirlnat]

yall imma bout to cry i really hate school

[danielaletelier]

Gracias por este materia! lo utilizo con mis estudiantes

[sujitkumar-wxbk]

Am eye macular degeneration pasent any treatment..?

[tarunraina]

I might be the only. Who is not watching this cause of school 😂 I was just curious about gene 🧬

[shivaschimera]

Could you imagine having to have small piece of your liver removed every month for 30 year's to study the development of liver cell's over long periods of time? Poor Poor slaves.

[cesarg]

This is an interesting video I’m leaving a comment do bio class from Cesar Gomez

[sokawai]

Im watching this cause yeah. Not because of school.
But umm knowing thats a school assignment...heheh bwoa, imma catch up with yall MWAHAHAHA🔥

[DanicaElle]

I wish we didn't test on animals.