CRISPR Gene Editing Prompts Chaos in DNA of Human Embryos
CRISPR Gene Editing Prompts Chaos in DNA of Human Embryos
Three studies identify unintended consequences of gene editing in human embryos, including large deletions and reshuffling of DNA.
CRISPR Gene Editing Prompts Chaos in DNA of Human Embryos
CRISPR Gene Editing Prompts Chaos in DNA of Human Embryos

Three studies identify unintended consequences of gene editing in human embryos, including large deletions and reshuffling of DNA.

Three studies identify unintended consequences of gene editing in human embryos, including large deletions and reshuffling of DNA.

gene therapy
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing
Abby Olena | Apr 10, 2020
Researchers slowed disease progression in the mice by injecting two different viral vectors, each containing one part of the DNA encoding the Cas9 protein, to edit the causative gene.
First Patient Receives In Vivo CRISPR Editing
First Patient Receives In Vivo CRISPR Editing
Jef Akst | Mar 4, 2020
Doctors in Oregon delivered the gene editing machinery behind the retina in hopes of treating an inherited form of blindness, according to the companies that developed the therapy.
Gene Therapy Finds a Fertile Home in Ohio
Gene Therapy Finds a Fertile Home in Ohio
Shawna Williams | Jan 13, 2020
The midwestern state has quietly laid the groundwork for a biotech hub.
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lottery Underway for Rare Muscle-Wasting Disease Gene Therapy
Lisa Winter | Jan 7, 2020
The announcement by Novartis, the maker of Zolgensma, has drawn mixed reactions from the spinal muscular atrophy community.
Dog Study Revives Concerns About Virus Used for Gene Therapy
Dog Study Revives Concerns About Virus Used for Gene Therapy
Jef Akst | Jan 6, 2020
Canines treated with an adeno-associated viral (AAV) vector showed evidence that the therapeutic DNA held within the virus can integrate into the host genome, risking the activation of oncogenes.
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Fetal Gene Therapy Helps Mice with Spinal Muscular Atrophy
Alejandra Manjarrez | Dec 11, 2019
The animals lived longer and showed milder symptoms than untreated mice, although they didn’t survive as long as wildtype mice.
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Trial of Gene Therapy for Duchenne Muscular Dystrophy Put on Hold
Catherine Offord | Nov 12, 2019
The US Food and Drug Administration halts a study by Solid Biosciences after a patient experiences severe side effects following treatment.
Google Bans Ads for Experimental Medical Treatments
Google Bans Ads for Experimental Medical Treatments
Ashley Yeager | Sep 9, 2019
The new policy will increase scrutiny and pressure on stem cell clinics, according to industry experts.
Leaders of AveXis Out Over Doctored Gene Therapy Data
Leaders of AveXis Out Over Doctored Gene Therapy Data
Kerry Grens | Aug 14, 2019
Novartis, which bought the biotech firm in 2018, announced that the company’s top scientists have left, and news reports say it’s because they were involved in using manipulated data to get the gene therapy Zolgensma approved.