A hybrid piggyBac AAV-transposon and LNP-transposase for treating arginase deficiency: A 100-fold dose reduction compared to AAV alone

Molecular Therapy 2026 Open access

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Authors: Rachel Xu, Taryn Diep, Kiran Singh, Stuti Bakshi, Grace F. Wu, Mimi Lu, Gloria Tang, Johannes Schwerk, Qin Sun, Johannes Häberle, Andreas Schulze, Ilona Tkachyova
+5 more Martin Poms, Bonnie E. Jacques, Joshua J. Rychak, Sang Hyeon Lee, Gerald S. Lipshutz
DOI: 10.1016/j.ymthe.2026.04.045
Cited by: 1
References: 54

Fields of Study & Subjects

Virus-based gene therapy research Neurogenetic and Muscular Disorders Research Parvovirus B19 Infection Studies

Publication details

Journal / source
Molecular Therapy
Publisher
Elsevier BV
Volume
34
Issue
8
Pages
4449–4465
Publication date
27 April 2026
Language
EN