hotspot -WSS

hotspot

author：focus 2025-09-27 23:53:36 - browse：32

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： Supreme Court strikes down use of affirmative action
Next article： Listen: Vivek Ramaswamy's star turn & leaky drug data

hotspot

WSS

Republicans love RFK Jr. What's that tell us about health in 2024?

Abortion policy in the spotlight at Republican debate

National associations that represent hospice owe an apology

Health officials lay out plans to cope with respiratory virus season

Study: Spinal cord stimulation helps patients with paralysis walk again

The United States is paying nearly triple for new Covid vaccines

entertainment

hotspot

entertainment

Walensky prepares to leave CDC — and Congress — behind

Congress extends some pandemic preparedness programs

Drug repurposing or repositioning? The language matters

Medicare innovation center doesn’t save money, experts say

At least 13 dead in Texas as scorching temps continue

Will an HIV vaccine ever be a reality?

Moonlake's readout produced a cash windfall. Risks remain

The United States is paying nearly triple for new Covid vaccines

Guidelines to prevent youth baseball injuries need more muscle

Congress extends some pandemic preparedness programs

knowledge

The biotech scorecard for Q4: 16 stock

With Roe v Wade overturned, abortion providers work to adapt

Abortion policy in the spotlight at Republican debate

How the first Native Hawaiian psychiatrist helped diversify medicine

DeSantis wants to import drugs. Biden’s FDA blasted his plan

How the first Native Hawaiian psychiatrist helped diversify medicine

hotspot

leisure time

focus

focus