Symptoms in ALS Mouse Model Improve with CRISPR Base Editing
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing
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.
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing

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.

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.

in vivo gene editing
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.
Researchers Launch First Study of In Vivo CRISPR Therapy in Humans
Researchers Launch First Study of In Vivo CRISPR Therapy in Humans
Catherine Offord | Jul 26, 2019
A trial is recruiting patients to test the gene-editing technology’s ability to treat an inherited form of blindness caused by a mutation in the CEP290 gene.
Preliminary Results Point to Success of In Vivo Gene Editing
Preliminary Results Point to Success of In Vivo Gene Editing
Carolyn Wilke | Feb 12, 2019
Two studies show signs that the introduced DNA is functioning, but it’s too early to know if patients actually benefit.
New Technique Limits CRISPR-Cas9 Off-Target Mutations
New Technique Limits CRISPR-Cas9 Off-Target Mutations
Abby Olena | Sep 12, 2018
A mouse study details a method called VIVO that predicts the accuracy of any guide RNA.
Early Results of First In Vivo Gene-Editing Trial Encouraging
Early Results of First In Vivo Gene-Editing Trial Encouraging
Kerry Grens | Sep 5, 2018
Two patients who received Sangamo’s zinc finger–based treatment for Hunter syndrome have lower biomarkers of the condition, but no signs of new enzyme production.