Last Updated: 09/26/2026
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Found 108 publications
Gene Editing, Peroxisomal Disorders, and Treatments for Rare Disease.
Journal: The New England journal of medicine
Published: August 12, 2026
Evaluating cases of possible insertional mutagenesis after AAV gene therapy.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: July 09, 2026
Neuroepithelial Tumor with AAV Integration after Intracisternal Magna Vector Delivery.
Journal: The New England journal of medicine
Published: May 13, 2026
Correction of a recurrent pathogenic variant in methylmalonic acidemia using adenine base editing.
Journal: bioRxiv : the preprint server for biology
Published: April 10, 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
The Need for Clinical Diagnostic Proficiency in the Genomic Era.
Journal: Pediatrics
Published: February 13, 2026
De novo MAP2K4 variants cause a novel neurodevelopmental syndrome with impaired JNK signaling in iPSC-derived neurons.
Journal: medRxiv : the preprint server for health sciences
Published: January 02, 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
Novel Features of RASopathies: Liver Disease as an Emerging Phenotype.
Journal: American journal of medical genetics. Part A
Published: October 13, 2025
How to create personalized gene editing platforms: Next steps toward interventional genetics.
Journal: American journal of human genetics
Published: October 09, 2025
Base editing strategies for in vivo correction of two highly recurrent phenylketonuria variants.
Journal: Molecular therapy. Nucleic acids
Published: September 08, 2025
Last Updated: 09/26/2026