explore -WSS

explore

author：focus 2025-10-01 23:02:44 - browse：44

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： Trump demands the U.S. pay no more for drugs than other countries … again
Next article： Bernie Sanders gets little from pharma CEOs on drug prices

Wikipedia

WSS

Why Eli Lilly’s earnings didn’t quite live up to the hype

Readout Newsletter: Replicate, Biogen's Leqembi, and Humira

Providers still hesitate to prescribe buprenorphine, study finds

Doctors on social media are being harassed and cyberbullied

Congress must protect the remaining independent doctors

7 of the most gruesome medical devices in history

focus

explore

entertainment

Anesthesiologist group: stop taking Ozempic before surgery

Juul documents reveal insider tactics on political lobbying

How one medical school became remarkably diverse

Our daughter's deadly disease: trisomy 18. We wanted to protect her

Psychedelics group wrestles with new pharma identity

Artificial intelligence use in health care faces greater scrutiny in 2024

Novavax promises a turnaround & Lilly roils the obesity market

Southern California school board OKs curriculum after Gov. Gavin Newsom threatened a $1.5M fine

Medicare proposal to cover medical devices draws a mixed reaction

Artificial intelligence advances fuel industry trying to preserve loved ones after death

entertainment

New rules on organ donation and transplantation 'will bring chaos'

Congress considering partial increase for doctor pay in Medicare

Lilly to acquire Dermira for $1.1 billion, adding eczema drug to its pipeline

Hyaluronic acid injections don’t help knee osteoarthritis, study finds

Southern California school board OKs curriculum after Gov. Gavin Newsom threatened a $1.5M fine

CDC backtracks, saying fully vaccinated people can go maskless indoors

knowledge

focus

Wikipedia

hotspot