explore -WSS

explore

author：focus 2025-09-03 15:26:19 - browse：3911

Anna Yeo/STAT

Recently approved gene therapies offer patients one-time, potentially curative treatments for genetic diseases such as sickle cell anemia and beta thalassemia. But “one-time” miracle solutions can often be multi-month affairs, require millions of dollars, and cause painful side effects. What if that doesn’t have to be the case?

In utero gene editing, or prenatal somatic cell genome editing, envisions treating a fetus diagnosed with a genetic disease before birth, thereby preventing that entire protocol and the onset of symptoms in the first place. It would also challenge the need for the ethically fraught enterprise of embryo editing, as the treatment would only make edits in the DNA of the individual fetus — edits which would not be passed on in a heritable way.

Watch this video to learn more about in utero gene editing, how it works, and why scientists believe it might be an advantageous approach to treating certain genetic diseases.

Previous article： Cancer drug shortages should be causing more outrage
Next article： Sarepta Duchenne therapy fails to meet primary endpoint in trial

WSS

US sending controversial cluster munitions to Ukraine until other ammo is ready: Kirby

After Sarepta trial failure, Duchenne families brace for more debate

Swim instructor mom warns parents not to buy blue swimsuits for kids

Sarepta Duchenne therapy fails to meet primary endpoint in trial

Inari Medical to buy amputation prevention company LimFlow

Kidney transplant patients stopped immunosuppressants in trial

entertainment

explore

Wikipedia

ChatGPT in medicine: STAT answers readers' burning questions

Brain injuries and the importance of a second opinion

Heart attack and stroke risk calculators need revising

Humans aren't the only ones with an imagination. Rats have it too

Medical leaders decry Supreme Court decision on affirmative action

See Ryan Reynolds, Hugh Jackman suited up in 1st look at 'Deadpool 3': Photo

Hollywood unions extend contract negotiations for actors

STAT Readout: The latest on Wegovy, Mounjaro, and Covid shots

Over 800 arrested across France in 3rd night of riots after fatal police shooting of teen

Pandemic and All Hazards Preparedness Act needs lean update

explore

Key Senate panel to consider PBM, drug pricing reform package

Readout LOUD podcast: The Sarepta saga, decoding biotech hype

How to convince healthy people to buy insurance

Wisconsin governor employs partial veto to fund schools for 4 centuries

Eisai exec who led development of Alzheimer’s drug to retire

DeSantis defends odds vs. Trump, despite early poll gap: 'We've worked really hard'

focus

Wikipedia

hotspot

leisure time