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JCI Insight|May 7, 2024
Potential limitations of microdystrophin gene therapy for Duchenne muscular dystrophyCora C Hart, Young Il Lee, Jun Xie, et al.
Human Gene Therapy|September 30, 2022
Direct ITR-to-ITR Nanopore Sequencing of AAV Vector GenomesSuk Namkung, Ngoc Tam Tran, Sangeetha Manokaran, et al.
Human Gene Therapy|February 11, 2025
Comprehensive Review of Osteogenesis Imperfecta: Current Treatments and Future InnovationsSachin Chaugule, Christodoulos Kypros Constantinou, Aijaz Ahmad John, et al.
Molecular Therapy. Nucleic Acids|August 7, 2023
Therapeutic inhibition of miR-155 attenuates liver fibrosis via STAT3 signalingShashi Bala, Yuan Zhuang, Prashanth Thevkar Nagesh, et al.
Viruses|June 28, 2023
Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time SequencingMitchell Yip, Jing Chen, Yan Zhi, et al.
JCI Insight|August 13, 2024
Systemic gene therapy corrects the neurological phenotype in a mouse model of NGLY1 deficiencyAiling Du, Kun Yang, Xuntao Zhou, et al.
Human Gene Therapy|September 10, 2013
Vector sequences are not detected in tumor tissue from research subjects with ornithine transcarbamylase deficiency who previously received adenovirus gene transferLi Zhong, Shaoyong Li, Mengxin Li, et al.
Genes & Development|December 18, 2020
A feed-forward regulatory loop in adipose tissue promotes signaling by the hepatokine FGF21Myoung Sook Han, Rachel J Perry, João-Paulo Camporez, et al.
Biomaterials|December 23, 2008
The targeting of 14-succinate triptolide-lysozyme conjugate to proximal renal tubular epithelial cellsZhirong Zhang, Qiang Zheng, Jing Han, et al.
EMBO Molecular Medicine|May 16, 2025
High-purity AAV vector production utilizing recombination-dependent minicircle formation and genetic couplingHao Liu, Nan Liu, Chen Zhou, et al.
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