Last Updated: 09/26/2026
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Found 60 publications
A hybrid piggyBac AAV-transposon and LNP-transposase for treating arginase deficiency: A 100-fold dose reduction compared to AAV alone.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: December 07, 2025
A hypomorphic model of CPS1 deficiency for investigating the effects of hyperammonemia on the developing nervous system.
Journal: Disease models & mechanisms
Published: January 25, 2025
Establishing a core outcome set for creatine transporter deficiency and guanidinoacetate methyltransferase deficiency.
Journal: Orphanet journal of rare diseases
Published: December 06, 2024
Establishing a Core Outcome Set for Creatine Transporter Deficiency and Guanidinoacetate Methyltransferase Deficiency.
Journal: medRxiv : the preprint server for health sciences
Published: October 07, 2024
Use of an oversized AAV8 vector for CPS1 deficiency results in long-term survival and ammonia control.
Journal: Molecular therapy. Nucleic acids
Published: August 08, 2024
Staying on target in gene and cell therapy.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: August 06, 2024
Recent developments in translational imaging of in vivo gene therapy outcomes.
Journal: Molecular therapy : the journal of the American Society of Gene Therapy
Published: July 31, 2024
A2/A2B Deceased Donor Kidney Transplantation Using A2 Titers Improves Access to Kidney Transplantation: A Single-Center Study.
Journal: Kidney medicine
Published: July 01, 2024
Last Updated: 09/26/2026