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Baby gets world's first personalised gene therapy treatment

Quotes

    • 88:88
      You Google 'CPS1 deficiency' and it's either fatality rate or liver transplant,
      The Sun
      the baby's mum, Nicole, said in a video released by Children's Hospital of Philadelphia (CHOP), where little KJ was treated
    • 88:88
      Joni L. Rutter
      As a platform, gene editing -- built on reusable components and rapid customization -- promises a new era of precision medicine for hundreds of rare diseases, bringing life-changing therapies to patients when timing matters most: Early, fast, and tailored to the individual,
      XinhuaNet
      said Joni L. Rutter, director of NIH's National Center for Advancing Translational Sciences
    • 88:88
      Years and years of progress in gene editing and collaboration between researchers and clinicians made this moment possible, and while KJ is just one patient, we hope he is the first of many to benefit from a methodology that can be scaled to fit an individual patient’s needs,
      The Telegraph
      said Dr Rebecca Ahrens-Nicklas, director of the Gene Therapy for Inherited Metabolic Disorders Frontier Programme (GTIMD) at the Children’s Hospital of Philadelphia
    • 88:88
      We’re still very much in the early stages of understanding what this medication may have done for KJ,
      CNN
      Ahrens-Nicklas said
    • 88:88
      As we get better and better at making these therapies and shorten the time frame even more, economies of scale will kick in and I would expect the costs to come down,
      CNN
      Musunuru said
    • 88:88
      We were, like, you know, weighing all the options, asking all the questions for either the liver transplant, which is invasive, or something that’s never been done before,
      CNN
      Nicole said
    • 88:88
      We prayed, we talked to people, we gathered information, and we eventually decided that this was the way we were going to go,
      CNN
      her husband added
    • 88:88
      This is the first step towards the use of gene editing therapies to treat a wide variety of rare genetic disorders for which there are currently no definitive medical treatments,
      CNN
      said Dr. Kiran Musunuru, a University of Pennsylvania gene editing expert who co-authored the study published Thursday in the New England Journal of Medicine
    • 88:88
      Once someone comes with a breakthrough like this, it will take no time” for other teams to apply the lessons and move forward
      CNN
      he said
    • 88:88
      Although this has been a very specific approach, partly motivated by the devastating nature of the disease, it represents a milestone that demonstrates these therapies are now a reality. As the article reports, the patient will be monitored for a long time to ensure his wellbeing and determine whether additional doses are needed to further improve the symptoms of the disease.”
      Guardian
      Dr Miguel Ángel Moreno-Mateos, a geneticist at Pablo de Olavide University, in Seville, said
    • 88:88
      The promise of gene therapy that we’ve heard about for decades is coming to fruition, and it’s going to utterly transform the way we approach medicine,
      Guardian
      said Prof Kiran Musunuru at the University of Pennsylvania
    • 88:88
      At every step of the process, we were always expecting someone to say, ‘No, sorry,
      The New York Times
      Dr. Kassim said
    • 88:88
      There was a lot of shooting from the hip,
      The New York Times
      Dr. Musunuru said
    • 88:88
      We loved him, and we didn’t want him to be suffering,
      The New York Times
      Ms. Muldoon said
    • 88:88
      At this point, the clock starts in my mind,
      The New York Times
      Dr. Musunuru said
    • 88:88
      Fyodor Urnov
      I don’t think this could have happened in any country other than the U.S.,
      The New York Times
      Dr. Urnov said
    • 88:88
      These steps traditionally take the better part of a decade, if not longer,
      The New York Times
      he said
    • 88:88
      One of the most terrifying moments was when I walked into the room and said, ‘I don’t know if it will work but I promise I will do everything I can to make sure it is safe,
      The New York Times
      she said