explore -WSS

explore

author：knowledge 2025-10-02 08:43:42 Page View：6525

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.

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

Previous article： Change Healthcare payments predicted restored March 15
Next article： Heart attack and stroke risk calculators need revising

leisure time

DIEP, the 'gold standard' of breast reconstruction, is under threat

AdobeIn1983,Iflewhomefromcollegetobewithmymotherasshewokeupfromamastectomy.Sheoptedoutofbreastrecons

STAT Summit: Biotech founders on deal

Fromleft,EliLilly'sJacobS.VanNaarden,Avenzo'sAthenaCountouriotis,andBiohaven'sVladCoriconstageatthe2

Hollywood unions extend contract negotiations for actors

1:36HollywoodwritersandtheirsupportersfromtheSAGAFTRAactors'unionwalkthepicketlineoutsideWarnerBrosS

Impact of Dobbs ruling felt at reproductive medicine meeting

MathieuLewis-Rolland/GettyImagesNEWORLEANS—TheannualmeetingoftheAmericanSocietyofReproductiveMedicin

explore

focus

leisure time

Impact of Dobbs ruling felt at reproductive medicine meeting