Phenotypic features of dystrophin gene knockout pigs harboring a human artificial chromosome containing the entire dystrophin gene

Generation of heterozygous PKD1 mutant pigs exhibiting early-onset renal cyst formation

(2022) Masahito Watanabe et al.   LABORATORY INVESTIGATION

Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled, phase 2 trial

(2022) Craig M McDonald et al.   LANCET

Current Pharmacological Strategies for Duchenne Muscular Dystrophy

(2021) Shanshan Yao et al.   Frontiers in Cell and Developmental Biology

A scalable, clinically severe pig model for Duchenne muscular dystrophy

(2021) Michael Stirm et al.   Disease Models & Mechanisms

Construction of stable mouse artificial chromosome from native mouse chromosome 10 for generation of transchromosomic mice

(2021) Satoshi Abe et al.   Scientific Reports

Transchromosomic technology for genomically humanized animals

(2020) Takashi Moriwaki et al.   EXPERIMENTAL CELL RESEARCH

Human artificial chromosomes for pluripotent stem cell-based tissue replacement therapy

(2020) Sergey A. Sinenko et al.   EXPERIMENTAL CELL RESEARCH

Somatic gene editing ameliorates skeletal and cardiac muscle failure in pig and human models of Duchenne muscular dystrophy

(2020) A. Moretti et al.   NATURE MEDICINE

Human AlphoidtetO Artificial Chromosome as a Gene Therapy Vector for the Developing Hemophilia A Model in Mice

(2020) Sergey V. Ponomartsev et al.   Cells

In vivo stem cell tracking using scintigraphy in a canine model of DMD

(2020) Inès Barthélémy et al.   Scientific Reports

Pluripotent stem cell-based gene therapy approach: human de novo synthesized chromosomes

(2020) Sergey A. Sinenko et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

Engineering of human induced pluripotent stem cells via human artificial chromosome vectors for cell therapy and disease modeling

(2020) Yasuhiro Kazuki et al.   Molecular Therapy-Nucleic Acids

Generation of H7N9-specific human polyclonal antibodies from a transchromosomic goat (caprine) system

(2019) Hua Wu et al.   Scientific Reports

Humanized UGT2 and CYP3A transchromosomic rats for improved prediction of human drug metabolism

(2019) Yasuhiro Kazuki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Dynamics of host and graft after cell sheet transplantation: Basic study for the application of amyotrophic lateral sclerosis

(2019) Mami Nakanishi et al.   BRAIN RESEARCH

Somatic Cell Nuclear Transfer Reprogramming: Mechanisms and Applications

(2018) Shogo Matoba et al.   Cell Stem Cell

Modeling lethal X-linked genetic disorders in pigs with ensured fertility

(2018) Hitomi Matsunari et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Timing and localization of human dystrophin isoform expression provide insights into the cognitive phenotype of Duchenne muscular dystrophy

(2017) Nathalie Doorenweerd et al.   Scientific Reports

A transgenic-cloned pig model expressing non-fluorescent modified Plum

(2016) Masaki NAGAYA et al.   JOURNAL OF REPRODUCTION AND DEVELOPMENT

Generation of heterozygous fibrillin-1 mutant cloned pigs from genome-edited foetal fibroblasts

(2016) Kazuhiro Umeyama et al.   Scientific Reports

Human artificial chromosomes for Duchenne muscular dystrophy and beyond: challenges and hopes

(2015) Francesco Saverio Tedesco   CHROMOSOME RESEARCH

How much dystrophin is enough: the physiological consequences of different levels of dystrophin in themdxmouse

(2015) Caroline Godfrey et al.   HUMAN MOLECULAR GENETICS

Production of transgenic cloned pigs expressing the far-red fluorescent protein monomeric Plum

(2015) Masahito WATANABE et al.   JOURNAL OF REPRODUCTION AND DEVELOPMENT

Use of a Human Artificial Chromosome for Delivering Trophic Factors in a Rodent Model of Amyotrophic Lateral Sclerosis

(2015) Yasuhiro Watanabe et al.   Molecular Therapy-Nucleic Acids

Low dystrophin levels in heart can delay heart failure in mdx mice

(2014) Maaike van Putten et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Concise Review: Mesoangioblast and Mesenchymal Stem Cell Therapy for Muscular Dystrophy: Progress, Challenges, and Future Directions

(2014) Suzanne E. Berry   Stem Cells Translational Medicine

An induced pluripotent stem cell-mediated and integration-free factor VIII expression system

(2013) Yuwna Yakura et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Highly stable maintenance of a mouse artificial chromosome in human cells and mice

(2013) Kanako Kazuki et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Dystrophin-deficient pigs provide new insights into the hierarchy of physiological derangements of dystrophic muscle

(2013) Nikolai Klymiuk et al.   HUMAN MOLECULAR GENETICS

Trans-chromosomic mice containing a human CYP3A cluster for prediction of xenobiotic metabolism in humans

(2012) Yasuhiro Kazuki et al.   HUMAN MOLECULAR GENETICS

Human Artificial Chromosomes for Gene Delivery and the Development of Animal Models

(2011) Yasuhiro Kazuki et al.   MOLECULAR THERAPY

Stem Cell-Mediated Transfer of a Human Artificial Chromosome Ameliorates Muscular Dystrophy

(2011) F. S. Tedesco et al.   Science Translational Medicine

Complete Genetic Correction of iPS Cells From Duchenne Muscular Dystrophy

(2009) Yasuhiro Kazuki et al.   MOLECULAR THERAPY

A highly Stable and Nonintegrated Human Artificial Chromosome (HAC) Containing the 2.4 Mb Entire Human Dystrophin Gene

(2008) Hidetoshi Hoshiya et al.   MOLECULAR THERAPY