When Rylae-Ann Poulin was 1 year old, she did not crawl or make noises like other kids her age. A rare genetic disease prevented her from even lifting her head. Her parents took turns holding her all night to ease her breathing and help her sleep peacefully. But months later, doctors in Taiwan treated the young patient’s brain directly, attacking the disease with genetic medicine. Now the 4-year-old is walking, running, swimming, reading and riding horses. Rylae-Ann lives with her family in Bangkok, Thailand. She was accepted into a medical trial for a new method of gene therapy. Experts say the treatment holds great promise for treating several brain diseases. The method was approved in Europe and the United Kingdom for a condition called AADC deficiency. The disorder damages communication systems among nervous system cells. American drug company PTC Therapeutics plans to seek U.S. approval for the treatment this year. The National Institutes of Health (NIH) says there are about 30 studies underway in the United States right now to test gene therapy to the brain. The studies target a number of diseases. Dr. Krystof Bankiewicz at Ohio State University leads one such study on AADC deficiency. Others test treatments for brain diseases like Alzheimer’s, Parkinson’s and Huntington’s. Difficulties remain, especially with diseases caused by more than a single gene. But scientists say evidence is growing in support of gene therapy to the brain. “There’s a lot of exciting times ahead of us,” said Bankiewicz, a brain surgeon. “We’re seeing some breakthroughs.”
Title: Gene Therapy Delivers Treatment Directly to Brain
Content: <div id='article-page'><div id='article-content' data-media-url='//news-app-staging.s3.amazonaws.com' data-base-url='//news-app-staging.herokuapp.com' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/A1.wav' data-capture-url='//staging.analytics.lingraphica.com/events/capture_news' data-article-title='Gene Therapy Delivers Treatment Directly to Brain' data-article-id='6365'><script src='//news-app-staging.herokuapp.com/javascripts/getscripts.js'></script><link rel='stylesheet' href='//news-app-staging.herokuapp.com/stylesheets/article.css' type='text/css' /><div class='article'><p><span data-start-time='0' data-end-time='8479'>When Rylae-Ann Poulin was 1 year old, she did not crawl or make noises like other kids her age.</span> <span data-start-time='8479' data-end-time='15333'>A rare genetic disease prevented her from even lifting her head. </span> <span data-start-time='15333' data-end-time='23417'>Her parents took turns holding her all night to ease her breathing and help her sleep peacefully.</span> <span data-start-time='23417' data-end-time='34583'>But months later, doctors in Taiwan treated the young patient’s brain directly, attacking the disease with genetic medicine. </span> <span data-start-time='34583' data-end-time='43167'>Now the 4-year-old is walking, running, swimming, reading and riding horses.</span> <span data-start-time='43167' data-end-time='47750'>Rylae-Ann lives with her family in Bangkok, Thailand. </span> <span data-start-time='47750' data-end-time='54542'>She was accepted into a medical trial for a new method of gene therapy. </span> <span data-start-time='54542' data-end-time='62583'>Experts say the treatment holds great promise for treating several brain diseases.</span> <span data-start-time='62583' data-end-time='71438'>The method was approved in Europe and the United Kingdom for a condition called AADC deficiency. </span> <span data-start-time='71438' data-end-time='78083'>The disorder damages communication systems among nervous system cells.</span> <span data-start-time='78083' data-end-time='88188'>American drug company PTC Therapeutics plans to seek U.S. approval for the treatment this year.</span> <span data-start-time='88188' data-end-time='101979'>The National Institutes of Health (NIH) says there are about 30 studies underway in the United States right now to test gene therapy to the brain. </span> <span data-start-time='101979' data-end-time='106333'>The studies target a number of diseases.</span> <span data-start-time='106333' data-end-time='116521'>Dr. Krystof Bankiewicz at Ohio State University leads one such study on AADC deficiency. </span> <span data-start-time='116521' data-end-time='124917'>Others test treatments for brain diseases like Alzheimer’s, Parkinson’s and Huntington’s.</span> <span data-start-time='124917' data-end-time='131958'>Difficulties remain, especially with diseases caused by more than a single gene. </span> <span data-start-time='131958' data-end-time='139333'>But scientists say evidence is growing in support of gene therapy to the brain.</span> <span data-start-time='139333' data-end-time='146938'>“There’s a lot of exciting times ahead of us,” said Bankiewicz, a brain surgeon. </span> <span data-start-time='146938' data-end-time='149604'>“We’re seeing some breakthroughs.”</span> </p></div><div class='control-buttons-sticky' style='display:none;'><div class='control-buttons'><button title='Back' class='back' disabled='disabled'></button><button title='Play' class='play' disabled='disabled'></button><button title='Pause' class='pause' style='display:none;'></button><button title='Forward' class='forward' disabled='disabled'></button><button class='finished-reading' style='display:none;'>Done</button></div></div></div><div id='article-media'><div id='media-image'><img src='https://news-app-production.s3.amazonaws.com/articles/5792/A1.jpeg'/></div></div><div id='question-content' style='display:none;'><div class='sentence_completion_question question-container' data-question-id=19198 data-hint-location='54542' style='display:none;'><div class='question' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/experts_say_the_treatment_holds_great_promise_for_treating_several__blank__diseases.wav'>Experts say the treatment holds great promise for treating several <span class='blank'>brain</span> diseases.</div><div class='choices' data-correct-choice='brain'><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/brain.wav'><span>brain</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/drain.wav'><span>drain</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/crane.wav'><span>crane</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/lane.wav'><span>lane</span></div></div></div><div class='vocabulary_question question-container' data-question-id=19199 data-hint-location='146938' style='display:none;'><div class='question' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/which_of_the_following_is_a_definition_of_the_word_breakthrough.wav'>Which of the following is a definition of the word breakthrough?</div><div class='choices' data-correct-choice='a sudden increase in knowledge, understanding, etc.'><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/a_sudden_increase_in_knowledge_understanding_etc.wav'><span>a sudden increase in knowledge, understanding, etc.</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/the_end_of_a_relationship.wav'><span>the end of a relationship</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/the_twohundredth_anniversary_of_a_significant_event.wav'><span>the two-hundredth anniversary of a significant event</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/a_person_who_rides_a_bicycle.wav'><span>a person who rides a bicycle</span></div></div></div><div class='multiple_choice_question question-container' data-question-id=19200 data-hint-location='88188' style='display:none;'><div class='question' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/according_to_the_national_institutes_of_health_roughly_how_many_studies_are_underway_in_the_us_to_test_gene_therapy_to_the_brain.wav'>According to the National Institutes of Health, roughly how many studies are underway in the U.S. to test gene therapy to the brain?</div><div class='choices' data-correct-choice='thirty'><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/thirty.wav'><span>thirty</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/forty.wav'><span>forty</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/fifty.wav'><span>fifty</span></div><div class='choice' data-sound-name='https://news-app-production.s3.amazonaws.com/articles/5792/sixty.wav'><span>sixty</span></div></div></div><div class='question-buttons'><button class='skip-button'></button><button class='hint-button'></button><button class='speak-button'></button></div></div></div>
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