Refugees are children. More than three million of them. How old are you guys . We visited refugee camps in jordan. Six . When it was 111 degrees. There was no Running Water, no indoor plumbing, but there was a brandnew sesame street, produced with characters that these Young Refugees can learn from, as so many kids in america have for years. ticking this musical prodigy has been performing around the world since he was 11 years old. And hes blind. Which caught the attention of this neuroscientist, who wanted to see what would happen inside his brain. Okay. Shall we give it a shot . Yep. The part thats used for sight when he listens to music. This is what changes in his brain. Jeez, lights up. ticking im lesley stahl. Im bill whitaker. Im anderson cooper. Im sharyn alfonsi. Im scott pelley. Those stories, tonight, on 60 minutes. ticking ticking stahl tonight, dr. Jon lapook on assignment for 60 minutes. Lapook 20 years ago, scientists stunned the world when they announced they had decoded the genes that make up a human being. They hoped to use that genetic blueprint to advance something called gene therapy which locates and fixes the genes responsible for different diseases. As we first reported last year, a Clinical Trial at the National Institutes of health is doing exactly that, in an attempt to cure sickle cell anemia a devastating genetic disease that kills hundreds of thousands of people around the world every year. For 15 months, we followed the scientists, and patients, who are ushering in a genetic revolution. Jennelle stephenson im excited. Ray stephenson today is the big day. Lapook its the day after christmas, 2017, and 27yearold Jennelle Stephenson has come with her father and brother from florida to the National Institutes of health, just outside washington, d. C. Jennelle stephenson good morning. Lapook shes one of a small group of patients to receive an infusion containing altered d. N. A. Nurse this is what they look like. Jennelle stephenson Merry Christmas to me. Brother best Christmas Present ever. Jennelle stephenson yay. Lapook the clear liquid in the bag contains jennelles stem cells, that have been genetically modified. Dr. Tisdale there are about 500 million in there. Jennelle stephenson oh, my goodness. Lapook the hope is the new d. N. A. In the cells will cure jennelle of sickle cell anemia a brutal disease that causes debilitating pain. At its worst, on a scale of zero to ten, how bad was your pain . Jennelle stephenson we can go beyond a ten. Its terrible, its horrible. Lapook pain where . Jennelle stephenson everywhere. My back, my shoulders, elbows, arms, legs, even my cheekbones, just pain. Lapook can you actually describe it . Jennelle stephenson its a very sharp, like, stabbing, almost feels like bonecrushing pain. Feels like someones kind of constricting your bones, and then releasing, constantly. Lapook pain from sickle cell can occur anywhere blood circulates. Thats because red blood cells, normally donutshaped, bend into an inflexible sickle shape, causing them to pile up inside blood vessels. The resulting traffic jam prevents the normal delivery of oxygen throughout the body, leading to problems that include bone deterioration, strokes and organ failure. The gene that causes sickle cell anemia evolved in places like subsaharan africa, because it protects people from malaria. There, millions have the disease, and its estimated more than 50 of babies born with it die before the age of five. Dr. Glassberg right on the bone there . Lapook in the united states, it affects 100,000 people mostly african americans. For jennelle, having the disease as a child often meant spending christmas in the hospital. As an adult, she struggled through pain to complete college, but keeping a job was tough because something as simple as walking up stairs could trigger a pain crisis. Do you have friends whove died from sickle cell . Jennelle stephenson i do. Yes, younger than me. Lapook and youve known this your whole life, growing up . Jennelle stephenson right. Lapook that you could potentially die early . Jennelle stephenson right. Yes. Lapook did you think you would die early . Jennelle stephenson i did, actually. When i hit about 22, i was like, you know, im for a sickle celler, im kind of middleaged right now. Lapook what are some of the things that youve always wanted to do that you couldnt do . Jennelle stephenson honestly, everybody laughs at me for this i just want to run, to be honest. Lapook things that most people would take for granted. Jennelle stephenson just basic things. Lapook one of the most cruel parts of the disease, jennelle and other patients have told us, is being accused of faking pain to get narcotics, being labeled a drugseeker. During one tri to the emergency department, when she fell to the floor in pain, a doctor refused to help her. Jennelle stephenson and im looking up at her, and im in tears, and, im like, im doing the best that i can. Lapook and ye thinking. , sometimedont liorry. Im in so much pain, and you think i just want some morphine. And it just makes me sad that some people in the medical Community Just dont get it. Dr. Francis collins so this would be my lab. Lapook dr. Francis collins is director of the National Institutes of health, the largest Biomedical Research agency in the world. He oversees a nearly 40 billion budget that funds more than 400,000 researchers worldwide. Clinton dr. Collins, please come up to the lectern. applause lapook dr. Collins was head of the human genome project at the n. I. H. In 2000 when he made a landmark announcement after a decade of work, scientists had finally decoded the genes that make up a human being. Dr. Collins today, we celebrate the revelation of the first draft of the human book of life. Lapook when did it all start for you . Dr. Collins i got excited about genetics as a firstyear medical student. A pediatric geneticist came to teach us about how genetics was relevant to medicine. And he brought patients to class, and one of the first patients he brought was a young man with Sickle Cell Disease who talked about the experience of sickle cell crises and how incredibly painful those are. And yet, it was all because of one single letter in the d. N. A. That is misplaced, a t that should have been an a. And that was profound. You could have all of that happen because of one letter that was misspelled. Lapook the double helix of d. N. A. Is made up of billions of pieces of genetic information. What dr. Collins is saying is. Out of all that, its just one error in the d. N. A. Code a t that should have been an a that causes sickle cell anemia. Fix that error, and you cure the disease. But figuring out how to do that would take more than 20 years of research. Do you remember when we used to sing lapook . And a little serendipity. Dr. Collins was playing in the n. I. H. Rock band in 2016 when his bass player hematologist dr. John tisdale started riffing on an idea. Dr. Tisdale wed finished setting up and went for a pizza, before dr. Collins i remember that. Dr. Tisdale before the gig. And at this point, i pitched to francis that it was really time that we do something definitive for Sickle Cell Disease. Lapook in the laboratory, dr. Tisdale and his collaborators created a gene with the correct spelling. Then, to get that gene into the patient, they used something with a frightening reputation h. I. V. , the virus that causes aids. It turns out, h. I. V. Is especially good at transferring d. N. A. Into cells. Dr. Tisdale so this shows the process. Lapook heres how it works. The corrected gene, seen here in yellow, is inserted into the h. I. V. Virus. Then, bone marrow stem cells are taken from a patient with sickle cell anemia. In the laboratory, those cells are combined with the virus carrying that new d. N. A. Dr. Tisdale this virus will then find its way to one of those cells and drop off a copy or two of the correctlyspelled gene. And then, these cells will go back to the patient. Lapook if the process works, the stem cells with the correct d. N. A. Will start producing healthy red blood cells. I can hear people, our viewers out there, thinking, wait a second, how do you know youre not going to get aids from the h. I. V. Virus . Dr. Tisdale the short answer is, we cut out the bits that cause infection in h. I. V. And we really replace that with the gene thats misspelled in Sickle Cell Disease, so that it transfers that instead of the infectious part. Lapook the stakes here are enormous. Dr. Collins yes. Lapook theres really very little safety net here, right . Dr. Collins make no mistake, were talking about very cuttingedge research, where the certainty about all the outcomes is not entirely there. We can look back at the history of gene therapy and see there have been some tragedies. Lapook deaths . Dr. Collins yes. Lapook in 1999, 18yearold Jesse Gelsinger received altered d. N. A. To treat a different genetic disease. He died four days later from a massive immune response. And in another trial, two children developed cancer. Jennelle stephenson understands this is a trial, with huge risks, and no guarantees. Jennelle stephenson this is oohearvethe n. I. H. Clinical cn december 2017, jennelle asked her brother, ray, for some help. Jennelle stephenson there goes ray, cutting my hair. Oh, snip. Lapook she decided to cut off all her hair, rather than watch it fall out from the massive dose of chemotherapy needed to suppress her immune rehe jcellowow to a leonal but. Im okay, it will grow back. Lapook a few days after the chemotherapy, jennelle received the infusion of genetically modified cells. Dr. Tisdale is it going good now . Nurse yes. Jennelle stephenson its just a waiting game. Lapook but the wait was a painful one. Not only for jennelle, but also for her father, ray. Ray stephenson let me fix this heating pad a little bit. Lapook . Who did what little he could as the effects of the chemotherapy kicked in, stripping jennelles throat and stomach of their protective layers. Jennelle stephnson oh, that hurts. Lapook she was unable to speak for a week, and lost 15 pounds. And, because having a severely weakened immune system means even a mild cold can turn deadly, jennelle had to stay in the hospital for nearly a month. After moving back to florida, she returned to the n. I. H. For periodic checkups. Dr. Tisdale these are her red blood cells. Lapook it didnt take long for dr. Tisdale to notice something was happening. This is jennelle before any treatment . Dr. Tisdale right. All across her blood you can see these really abnormal shapes. This one in particular is shaped like a sickle. Lapook nine months later, this is what dr. Tisdale saw not a sickle cell in sight. Was there ever a moment where you saw one of these normal looking smears and thought, is this the right patient . Dr. Tisdale oh, absolutely. When youre a scientist, youre skeptical all the time. So, first thing you do is look and make sure its that patient, go grab another one, make sure its the same. And weve done all that. And, indeed, her blood looks normal. Teacher move. Switch your arms and move. Lapook remember, jennelle used to struggle just to walk up a flight of stairs. Teacher and you fall. Lapook . And a fall like this would have landed her in the hospital. Teacher boom. Yeah. Good job, you did it. Bam. Lapook jennelle. You look amazing. Jennelle stephenson thank you lapook i have to say, i was a little nervous when you were thrown and you went down on the mat. Jennelle stephenson it was nothing. It was nothing. My body just felt strong. Lapook tell me about the adjustment that you need to make, to go from the old you to the new you. Jennelle stephenson my body, it almost felt like it was, like, itching to do more. And i was like, all right, well, lets go swimming today. Lets go to the gym today. Im like, all right, my body loves this. I kind of like it because my, i guess, all my endorphins started pumping. Lapook the endorphin high, something you had never experienced. Jennelle stephenson never experienced before. Yep. Pthrough your head as you were watching jennelle being thrown down to the mat . Ray stephenson i was just saying, thank you, lord. Thank you for medical science. And thank you for giving r w li Jennelle Stephenson new life, indeed. Lynndrick holmes ive never lived before. Lapook 18 other adults with sickle cell anemia have undergone the same gene therapy all responded well, except one who died from another cause. Dr. Francis collins says it will take years to improve the treatment so it can be made more widely available. Dr. Collins heres another dream. There are 7,000 genetic diseases for which we know the precise d. N. A. Misspelling. Couldnt this same strategy, this same set of principles work for lots of those, maybe someday all of them . Lapook youve been working on this for decades. Youre at a moment which is significant. Dr. Collins to lead the human genome project and to put that foundation in place. And now, to see that emerging not just as hopedfor advances, but real data showing cures for people. Lapook you just used the curing word. laughs youre willing to say that . Dr. Collins i believe that e. Ive got to be careful. But from every angle that i know how to size this up, this looks like a cure. ticking stahl there are more People Living as refugees around the world today than at any time since the second world war. And with conflicts dragging on for years, being a refugee now often means not going home for decades. Thats literally a lifetime, for millions of Young Children. As we first reported last fall, the refugee crisis has sparked a partnership between two of the countrys leading Nonprofit InstitutionsSesame Workshop, creator of sesame street, and the International Rescue committee the i. R. C. A Refugee Assistance Organization originally founded by albert einstein. For 50 years, sesame street has been teaching Young Children that one plus one equals two; but by teaming up with the i. R. C. To help the youngest refugees, its hoping that one plus one can now add up to farme this is the zaatari refugee it houses 77,000 syrian refugees. This is azraq camp. It houses another 35,000. Its hard to fathom that these tens of thousands are just a fraction of the more than six million syrians now living as refugees most of them for the last four to eight years. And nearly half of all of them are children. So this is the big waterhole. The first thing we noticed when we arrived at azraq camp with our guide, Laila Hussein of the i. R. C. , was the kids. Little kids carrying the water. Laila hussein yeah. Stahl theres no Running Water here, no indoor plumbing. Toilets are outside and shared by six families. The day we visited it was 111 degrees. Ask these young fellows if they come to get water every day. Hussein they carry the water. Stahl you carry . How old are you guys . How old . Stahl six. Eight meeting the needs of kids like this. Six. Is not what the humanitarian aid system was set up for. David miliband the humanitarian sector has prided itself on keeping people alive. Stahl David Miliband is head of the International Rescue committee. Traditionally, i guess that refugees flee war, go home when the war ends. Miliband yeah, the theory is that you just keep people alive until they can go home. But we know now that the average length of displacement for a refugee is close to 20 years stahl 20 years . Miliband close to 20 years. And thats why its a total tragedy that less than 2 of all humanitarian aid funding goes on education, even though half of the worlds refugees are kids. Stahl and only a tiny sliver of that 2 goes to educating Young Children. Miliband and thats a problem, because we know that its the earliest years that count the most. Staho one knows the importance of those earliest years and how to reach, and teach, kids in them better than sesame street. 8, 9, 10, 11. Stahl its been using television to educate kids in the u. S. , including tackling tough subjects like racism and death, for five decades. And its done local versions in other countries. In 2016, Sesame Workshop and the i. R. C. Had been strategizing about how they could collaborate to help refugee children, when a new competition was announced. The Macarthur Foundation is launching a new competition. Stahl the prize a stunning 100 million. Miliband the Macarthur Foundation offered 100 million to any organization who was ready to, solve a big global problem. Stahl a global problem that was intractable. Miliband we defined the global problem we wanted to tackle was trauma, toxic stress among refugee children in the middle east. We can reach literally millions of children. Stahl in the final pitch to the competitions judges, miliband and his Sesame Workshop counterpart sherrie westin, presented a twopronged plan Sesame Workshop would create a new show for the middle east. Sherrie westin we will introduce new local muppets. Stahl and the i. R. C. Would dramatically expand its services to Young Refugee kids directly, including where theyre living. And they gave you 100 million. Miliband yeah, 100 million is not as much as it sounds. Because its stahl it isnt . Miliband its over stahl it sounds huge. Miliband its over laughs five years. And were delivering inperson services to over a million kids and educational content via tv to nearly eight million kids. So its a big enterprise. Stahl production of the new television show, ahlan simsim, meaning welcome sesame, is well underway in jordan. It stars a spunky purple girl muppet named basma, a boy muppet, jad, who has just moved into the neighborhood, and their pal and comedic sidekick, a mischievous baby goat. Jad isnt labeled a refugee on the show, but there are hints. Hes voiced by a syrian puppeteer, and in this episode, where the other characters are showing each ot