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News Articles: gene therapy

KJ Muldoon, who was born at Children's Hospital of Philadelphia in August, reaches out to doctors after being treated for a rare genetic disorder using CRISPR technology.

Tagged as: 

  • Health

A promising genetic treatment tailor-made for a baby born with a rare disorder

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as CRISPR to treat a baby with a rare, life-threatening genetic disorder.

May 15, 2025
|
By:
  • Rob Stein
As his stem cells are collected, DeShawn “DJ” Chow talks about his upcoming sickle cell gene therapy treatment with pediatric hematologist-oncologist Leo Wang at City of Hope Children’s Cancer Center on May 22.

Tagged as: 

  • Health

Sickle cell gene therapies roll out slowly

It’s been almost a year since the Food and Drug Administration approved the first genetic treatments for sickle cell disease. So far, only a few patients have received the long-awaited treatments.

September 19, 2024
|
By:
  • Rob Stein
An experimental gene therapy tested in young children with an inherited form of deafness restored some hearing for most of them.

Tagged as: 

  • Health

Gene therapy shows promise for an inherited form of deafness

Scientists report that gene therapy restored at least some hearing and speech for five out of six children with a rare form of genetic deafness.

January 25, 2024
|
By:
  • Rob Stein
Preliminary results from a study show that gene-editing technology can be used to successfully treat a genetic disorder that increases the risk of heart disease.

Tagged as: 

  • Health

For the first time, gene-editing provides hints for lowering cholesterol

Researchers report the first results from a study testing the revolutionary gene-editing technique known as CRISPR for cutting high cholesterol.

November 13, 2023
|
By:
  • Rob Stein
"It's really life-changing," says Victoria Gray, when describing the gene-editing treatment for sickle cell disease that she received as part of a clinical trial in 2019.

Tagged as: 

  • Health

FDA advisers see no roadblocks for gene-editing treatment for sickle cell disease

Advisers to the Food and Drug Administration meeting Tuesday paved the way for the first treatment of human disease using the gene-editing technique CRISPR. The agency has a December deadline.

October 31, 2023
|
By:
  • Rob Stein
The Food and Drug Administration approved the first gene therapy to treat the most common form of muscular dystrophy.

Tagged as: 

  • Health

Muscular dystrophy patients get first gene therapy

The Food and Drug Administration approved the first gene therapy for Duchenne muscular dystrophy, but limited access to those ages four and five.

June 22, 2023
|
By:
  • Rob Stein
He Jiankui announced nearly five years ago that he had created the first gene-edited babies.

Tagged as: 

  • World

His baby gene editing shocked ethicists. Now he's in the lab again

He Jiankui, who shocked the world in 2018 by announcing the creation of the first gene-edited babies, tells NPR he's now working on a cure for Duchenne muscular dystrophy.

June 08, 2023
|
By:
  • John Ruwitch
Susan and Chris Finazzo have enrolled their sons Dylan and Chase in a study of gene therapy for Duchenne muscular dystrophy. The experimental treatment is still being studied but researchers hope it may help prevent the devastating effects of the disease.

Tagged as: 

  • Health

Gene therapy for muscular dystrophy stirs hopes and controversy

The FDA is considering greenlighting the experimental treatment under its accelerated approvals program. Some critics point out the therapy isn't yet proven to work and may be costly.

May 02, 2023
|
By:
  • Rob Stein
Researchers meeting in London this week concluded that techniques that have made it easier to manipulate DNA still produce too many mistakes for scientists to be confident any children born from edited embryos (such as these, photographed in 2018) would be healthy.

Tagged as: 

  • Health

Ethical concerns temper optimism about gene-editing for human diseases

The Third International Summit on Genome Editing concluded Monday with ethicists warning scientists to slow down efforts to use gene-editing to enhance the health of embryos.

March 10, 2023
|
By:
  • Rob Stein
Carlene Knight, 54, is one of the first patients in a landmark study designed to try to restore vision in those who have a rare genetic disease that causes blindness.

Tagged as: 

  • Health

Blind Patients Hope Landmark Gene-Editing Experiment Will Restore Their Vision

The unprecedented study involves using the gene-editing technique CRISPR to edit a gene while it's still inside a patient's body. In exclusive interviews, NPR talks with two of the first participants.

May 10, 2021
|
By:
  • Rob Stein
Conner Curran, 9, (right) and his brother Will, 7, at their home in Ridgefield, Conn., this week. The gene therapy treatment that stopped the muscle wasting of Conner's muscular dystrophy two years ago took more than 30 years of research to develop.

Tagged as: 

  • Health

A Boy With Muscular Dystrophy Was Headed For A Wheelchair. Then Gene Therapy Arrived

Gene therapy has helped a 9-year-old boy regain enough muscle strength to run. If successful in others, the treatment could change the lives of thousands of children with Duchenne muscular dystrophy.

July 27, 2020
|
By:
  • Jon Hamilton
Biomarin Pharmaceutical, a California company that makes what could become the first gene therapy for hemophilia, says its drug's price tag might be $3 million per patient.

Tagged as: 

  • Health

Gene Therapy Shows Promise For Hemophilia, But Could Be Most Expensive U.S. Drug Ever

The first gene therapy for hemophilia could be approved by the FDA within six months, according to the drugmaker, raising hopes among families. But the drug's price could be $3 million per patient.

July 20, 2020
|
By:
  • Rob Stein

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