crispr

For kids with rare genetic disorders, customized CRISPR treatments offer hope

6:00am December 13, 2024

The gene-editing technique is effective for treating some illnesses but it's been too expensive to consider it for rare conditions. A new approach in the works could make it more widely available.

"The therapy has really transformed my life more than I could have ever imagined," Victoria Gray, the first person to receive the CRISPR gene-editing treatment tellls NPR. "It gave me a new lease on life."

Health & Safety

Sickle cell patient's journey leads to landmark approval of gene-editing treatment

5:00am December 25, 2023

by Rob Stein

In early December, the Food and Drug Administration approved a gene-editing treatment for sickle cell disease, the first for any illness. One patient helped pave the way.

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.

Health & Safety

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

4:09pm November 12, 2023

by Rob Stein

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

"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.

Health & Safety

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

8:30pm October 31, 2023

by Rob Stein

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.

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Scientists have engineered an albino squid that provides a window into the inner workings of the brain.

Health & Safety

How scientists engineered a see-through squid with its brain in plain view

12:13pm August 28, 2023

by Jon Hamilton

A see-through squid is giving scientists an unprecedented view of the brain and other organs in a living animal.

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Mosquitoes spread malaria. Now researchers hope that a gene drive technology could turn them into malaria fighters. Although not every scientist thinks it's a good idea to genetically modify a wild animal.

Mosquitoes spread malaria. These researchers want them to fight it instead

5:05am July 20, 2023

by Geoff Brumfiel

Scientists have used a gene-editing technique to make mosquitos allies in the fight against malaria. Environmentalists are troubled by the idea of genetically modifying wild animals.

He Jiankui announced nearly five years ago that he had created the first gene-edited babies.

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

1:03pm June 08, 2023

by John Ruwitch

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.

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In London to address a gene-editing summit last week, Victoria Gray took a break to visit Sir John Soane's Museum. In 2019, Gray became the first patient to be treated for sickle cell disease using CRISPR, an experimental gene-editing technique. She was invited to talk about her experiences at the Third International Summit on Human Genome Editing.

Sickle cell patient's success with gene editing raises hopes and questions

1:37pm March 16, 2023

by Rob Stein

A Mississippi woman's life has been transformed by a treatment for sickle cell disease with the gene-editing technique CRISPR. All her symptoms from a disease once thought incurable have disappeared.

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.

Ethical concerns temper optimism about gene-editing for human diseases

5:27pm March 08, 2023

by Rob Stein

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.

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Biophysicist He Jiankui addressed the last international summit on human genome editing in Hong Kong in 2018. His experiments in altering the genetic makeup of human embryos was widely condemned by scientists and ethicists at the time, and still casts a long shadow over this week's summit in London.

Experts weigh medical advances in gene-editing with ethical dilemmas

3:12pm March 06, 2023

by Rob Stein

The last time this summit convened in 2018, the world was shocked to hear a scientist had created the first gene-edited babies. He was condemned, but gene-editing has continued, with some success.

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