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Gene, rare diseases

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

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.

Healio · 4d

Infant with rare disease receives world’s first personalized gene-editing therapy

An infant with a rare urea cycle disorder became the first patient to receive a personalized gene-editing therapy. His care team developed the one-of-a-kind medication in just 6 months. By the second day of his life, Kyle and Nicole Muldoon knew their newborn son was very sick.

Public Broadcasting Service (PBS)4d

Breakthrough gene editing treatment helps child born with rare disorder

Doctors announced this week that they have treated a newborn baby with a rare genetic disease using the world’s first personalized gene editing therapy. Geoff Bennett discussed the treatment and its ...

BioSpace13d

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 ...

STAT4d

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 ...

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