<code id='1F71C245FF'></code><style id='1F71C245FF'></style>
    • <acronym id='1F71C245FF'></acronym>
      <center id='1F71C245FF'><center id='1F71C245FF'><tfoot id='1F71C245FF'></tfoot></center><abbr id='1F71C245FF'><dir id='1F71C245FF'><tfoot id='1F71C245FF'></tfoot><noframes id='1F71C245FF'>

    • <optgroup id='1F71C245FF'><strike id='1F71C245FF'><sup id='1F71C245FF'></sup></strike><code id='1F71C245FF'></code></optgroup>
        1. <b id='1F71C245FF'><label id='1F71C245FF'><select id='1F71C245FF'><dt id='1F71C245FF'><span id='1F71C245FF'></span></dt></select></label></b><u id='1F71C245FF'></u>
          <i id='1F71C245FF'><strike id='1F71C245FF'><tt id='1F71C245FF'><pre id='1F71C245FF'></pre></tt></strike></i>

          explore

          explore

          author:explore    Page View:29465
          Terry Horgan pictured at home -- coverage from STAT
          Terry Horgan in his family's Montour Falls, N.Y., home, in an undated photo. Kate Collins/The Journal via AP

          Last October, a 27-year-old man with Duchenne muscular dystrophy died after receiving a CRISPR-based treatment custom built to treat his particular genetic mutation.

          Results of a detailed investigation released on Wednesday suggest that the patient, Terry Horgan, likely died of a previously undocumented adverse effect of the virus used to deliver the CRISPR machinery to his cells.

          advertisement

          These viruses, known as adeno-associated viruses, or AAVs, are used in several approved drugs and hundreds of clinical trials to deliver genes into patients, because they are generally benign. But at high doses they can cause side effects and, occasionally, deaths through one of several mechanisms, such as directly injuring the liver or triggering a type of immune response known as complement activation.

          Get unlimited access to award-winning journalism and exclusive events.

          Subscribe Log In

          focus

          Heart attack and stroke risk calculators need revising
          Heart attack and stroke risk calculators need revising

          AdobeForbusyprimarycarephysicianslikeme,anannualphysicalwitha27-year-oldmaleisablessing.Sincewe’real

          read more
          What happened to the House vote on health price transparency?
          What happened to the House vote on health price transparency?

          AlexHogan/STATYou’rereadingthewebeditionofD.C.Diagnosis. Signuphere toreceivethisnewsletterinyourinb

          read more
          In age of alternative facts, a scholarly course on calling out crap
          In age of alternative facts, a scholarly course on calling out crap

          Screenshotviacallingbullshit.orgTiredofalternativefacts,fakenews,andbreathlesshyperbole,twoprofessor

          read more

          Readout LOUD podcast, live from the STAT Breakthrough Summit East

          Isthereacureforallergies?HastheFDAbecometooflexible?Andwhichdrugsmakeyoumuscular?Wecoverallthatandmo