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Gene, Doctors and rare diseases

Smithsonian Magazine · 6h

In a Remarkable First, a Baby With a Rare Disease Receives Personalized Gene Therapy

Researchers say the CRISPR-based technique used could eventually be employed to treat more people with rare genetic diseases

People on MSN · 3d

Doctors Achieve Medical Breakthrough Using Gene-Editing Therapy to Heal Baby with Rare Disorder

Doctors have successfully used gene-editing therapy to treat an infant born with a rare disorder. Experts believe the breakthrough treatment could help many others with rare genetic diseases, a Children's Hospital of Philadelphia news release reported.

ABC News on MSN · 4d

Doctors save baby's life with first-ever gene fix for deadly rare disease

The goal is to reuse key parts of the treatment and simply swap in a custom set of instructions for each patient's specific gene mutation, doctors say.

At gene therapy meeting, no one wants to talk about the Vinay Prasad (not) in the room

At the American Society of Cell and Gene Therapy conference, there were questions and qualms on how Vinay Prasad may change ...

4don MSN

Gene editing helped a desperately ill baby thrive. Scientists say it could someday treat millions

The baby, KJ Muldoon of Clifton Heights, Penn., is one of 350 million people worldwide with rare diseases, most of which are ...

CRISPR’s Casgevy on the Rise With More Gene Therapy Proof of Concept To Come in 2025

CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy ...

Endpoints News13d

CRISPR Therapeutics' gene editing therapy reduces bad cholesterol and triglycerides by as much as 80%

CRISPR Therapeutics has revealed promising but early data from its first clinical study of a gene editing therapy to lower ...

5d

This baby's future was bleak. Then he became a medical first.

KJ Muldoon was born with a devastating medical problem. Racing to solve it, doctors may have launched a 'roadmap' for a new ...

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