Last Updated: 09/26/2026
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Found 178 publications
Correction of a recurrent pathogenic variant in methylmalonic acidemia using adenine base editing.
Journal: bioRxiv : the preprint server for biology
Published: April 10, 2026
Gene Editing Therapy in Cardiovascular Disease: 2026 ACC Scientific Statement: A Report of the American College of Cardiology.
Journal: Journal of the American College of Cardiology
Published: March 26, 2026
Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platform.
Journal: American journal of human genetics
Published: March 24, 2026
ASGCT's 2025 breakthroughs in targeted in vivo gene editing: Meeting summary and insights.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: January 12, 2026
The dawn of interventional genetics.
Journal: American journal of human genetics
Published: January 09, 2026
In vivo adenine base editing rescues brain biochemistry and improves motor deficits in a common phenylketonuria variant.
Journal: Molecular therapy. Advances
Published: December 17, 2025
Response to the FDA's proposed pathway for individualized genetic therapies.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: December 10, 2025
Efficient prime editing in vivo and in vitro using lipid nanoparticles.
Journal: Nature nanotechnology
Published: October 23, 2025
How to create personalized gene editing platforms: Next steps toward interventional genetics.
Journal: American journal of human genetics
Published: October 09, 2025
Liver-directed base editing of ABCC6 prevents ectopic calcification in a variant-humanized mouse model of pseudoxanthoma elasticum.
Journal: Molecular therapy. Nucleic acids
Published: September 27, 2025
Breakthroughs in targeted in vivo gene editing: Overcoming barriers to effective delivery.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: September 25, 2025
Last Updated: 09/26/2026