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On the Hunt for the Next Breakthrough in Motor Neuron Disease
On the Hunt for the Next Breakthrough in Motor Neuron Disease
Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.
Researchers leave no stone unturned in the search for a spinal muscular atrophy treatment strategy that uses base editing.
in vivo gene editing
Targeting a Genetic Accident to Treat Disease
Deanna MacNeil, PhD | May 9, 2023 | 2 min read
David Liu shares how integrating chemistry and evolution in his research has directed his work on base editing techniques aimed at developing new therapeutics.
New CRISPR Technique Causes Few Unintended Mutations in Fruit Flies
Jason P. Dinh | Jul 1, 2022 | 4 min read
A study finds that CRISPR-Nickase, which changes just one allele of a given gene, improves gene editing efficiency compared with CRISPR-Cas9.
Injecting Cockroaches with CRISPR Gene Edits Their Offspring
Sophie Fessl, PhD | May 25, 2022 | 4 min read
A new method has allowed researchers to conduct the first gene knock-out and knock-in edits on cockroaches and may extend to many other insects.
Early Successes Make CRISPR-Based Medicine a Possibility
Aparna Nathan, PhD | Oct 25, 2021 | 4 min read
CRISPR-Cas9’s molecular scissors—thus far limited to the lab bench—may soon find themselves at work directly in the human body.
Panel Lays Out Guidelines for CRISPR-Edited Human Embryos
Lisa Winter | Sep 4, 2020 | 2 min read
The International Commission on the Clinical Use of Human Germline Genome Editing claims the technology is still too risky for therapeutic use.
CRISPR Gene Editing Prompts Chaos in DNA of Human Embryos
Amanda Heidt | Jun 26, 2020 | 3 min read
Three studies identify unintended consequences of gene editing in human embryos, including large deletions and reshuffling of DNA.
Symptoms in ALS Mouse Model Improve with CRISPR Base Editing
Abby Olena, PhD | Apr 10, 2020 | 4 min read
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
Jef Akst | Mar 4, 2020 | 3 min read
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
Catherine Offord | Jul 26, 2019 | 1 min read
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
Carolyn Wilke | Feb 12, 2019 | 2 min read
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
Abby Olena, PhD | Sep 12, 2018 | 3 min read
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
Kerry Grens | Sep 5, 2018 | 2 min read
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.