Nature Search

Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

In vivo delivery of CRISPR-Cas9 holds promise for treating muscular dystrophy, however, AAV delivery is known to be immunogenic. Here, the authors show that LNP delivery of CRISPR-Cas9 enables repeated injections into skeletal muscle and leads to restored dystrophin expression in multiple muscle groups.

Eriya Kenjo
Hiroyuki Hozumi
Akitsu Hotta
ResearchOpen Access08 Dec 2021
Nature Communications

Volume: 12, P: 1-13
CRISPR-Cas3 induces broad and unidirectional genome editing in human cells

Class 1 CRISPR systems are not as developed for genome editing as Class 2 systems are. Here the authors show that Cas3 can be used to generate functional knockouts and knock-ins, as well as Cas3-mediated exon-skipping in DMD cells.

Hiroyuki Morisaka
Kazuto Yoshimi
Tomoji Mashimo
ResearchOpen Access06 Dec 2019
Nature Communications

Volume: 10, P: 1-13
Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

Expression of Cas9 and gRNA from viral vectors in vivo may cause off-target activity. Here the authors present NanoMEDIC, which uses nanovesicles to transiently deliver editing machinery to hard-to-transfect cells.

Peter Gee
Mandy S. Y. Lung
Akitsu Hotta
ResearchOpen Access13 Mar 2020
Nature Communications

Volume: 11, P: 1-18
An iPSC-based in vitro model recapitulates human thymic epithelial development and multi-lineage specification

Thymic epithelial cells (TECs) are essential for T cell development but how they develop from progenitors in humans is difficult to study. Here authors set up an in vitro system for thymus organogenesis in which induced pluripotent stem cells give rise to mature and diverse TECs that fully support T cell differentiation.

Yann Pretemer
Yuxian Gao
Yoko Hamazaki
ResearchOpen Access25 Aug 2025
Nature Communications

Volume: 16, P: 1-23
Optimization of the proliferation and persistency of CAR T cells derived from human induced pluripotent stem cells

Cytotoxic CAR T cells derived from human induced pluripotent stem cells can be genetically engineered for enhanced proliferation and persistency in solid tumours.

Tatsuki Ueda
Sara Shiina
Shin Kaneko
ResearchOpen Access12 Dec 2022
Nature Biomedical Engineering

Volume: 7, P: 24-37
Generation of hypoimmunogenic T cells from genetically engineered allogeneic human induced pluripotent stem cells

T cells derived from human induced pluripotent stem cells lacking certain components of the human leukocyte antigen system and incorporating a ligand that inhibits natural killer cells escape rejection when implanted in allogeneic mice.

Bo Wang
Shoichi Iriguchi
Shin Kaneko
Research17 May 2021
Nature Biomedical Engineering

Volume: 5, P: 429-440
Cartilage tissue engineering identifies abnormal human induced pluripotent stem cells

Akihiro Yamashita
Shiying Liu
Derrick E. Rancourt
ResearchOpen Access13 Jun 2013
Scientific Reports

Volume: 3, P: 1-6
Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency

Lentiviral vectors that express a fluorescent reporter and a selectable marker in pluripotent cells improve and simplify isolation of induced pluripotent stem cell lines in mouse and human.

Akitsu Hotta
Aaron Y L Cheung
James Ellis
Research26 Apr 2009
Nature Methods

Volume: 6, P: 370-376
Genetically engineered hypoimmunogenic cell therapy

To overcome the immune rejection issue in allogeneic cell transplants, various genetic engineering strategies have been developed. This Review explores these advances and discusses challenges in evading immune responses, as well as considerations for clinical development.

Akitsu Hotta
Sonja Schrepfer
Andras Nagy
Reviews29 Jul 2024
Nature Reviews Bioengineering

Volume: 2, P: 960-979
Srf destabilizes cellular identity by suppressing cell-type-specific gene expression programs

The transcription factor Srf is a central regulator of immediate-early and actin cytoskeletal genes. Here the authors show that Srf is activated by repression of β-actin, promoting iPSC reprogramming of neural progenitor cells and hepatoblasts by repressing cell-type specific genes.

Takashi Ikeda
Takafusa Hikichi
Shinji Masui
ResearchOpen Access11 Apr 2018
Nature Communications

Volume: 9, P: 1-15
Site-specific randomization of the endogenous genome by a regulatable CRISPR-Cas9 piggyBac system in human cells

Kentaro Ishida
Huaigeng Xu
Akitsu Hotta
ResearchOpen Access10 Jan 2018
Scientific Reports

Volume: 8, P: 1-12
Optimized simple culture protocol for inducing mature myotubes from MYOD1-overexpressed human iPS cells

Eiji Wada
Nao Susumu
Yukiko K. Hayashi
ResearchOpen Access20 Nov 2024
Scientific Reports

Volume: 14, P: 1-12
EOS lentiviral vector selection system for human induced pluripotent stem cells

Akitsu Hotta
Aaron Y L Cheung
James Ellis
Protocols03 Dec 2009
Nature Protocols

Volume: 4, P: 1828-1844
CD151 expression marks atrial- and ventricular- differentiation from human induced pluripotent stem cells

The authors demonstrate that CD151 expression distinguishes atrial from ventricular cardiomyocytes derived from induced pluripotent stem cells, while further showing the role of Notch signaling in mediating the process.

Misato Nakanishi-Koakutsu
Kenji Miki
Yoshinori Yoshida
ResearchOpen Access28 Feb 2024
Communications Biology

Volume: 7, P: 1-14
iPSC-derived type IV collagen α5-expressing kidney organoids model Alport syndrome

The development of a kidney organoid model from induced pluripotent stem cells (iPSCs) recapitulates collagen composition from mild and severe cases of Alport syndrome, establishing a kidney organoid model aiding drug discovery.

Ryuichiro Hirayama
Kosuke Toyohara
Kenji Osafune
ResearchOpen Access28 Sept 2023
Communications Biology

Volume: 6, P: 1-11
Establishment of quantitative and consistent in vitro skeletal muscle pathological models of myotonic dystrophy type 1 using patient-derived iPSCs

Ryu Kawada
Tatsuya Jonouchi
Hidetoshi Sakurai
ResearchOpen Access11 Jan 2023
Scientific Reports

Volume: 13, P: 1-17
Calcium dysregulation contributes to neurodegeneration in FTLD patient iPSC-derived neurons

Keiko Imamura
Naruhiko Sahara
Haruhisa Inoue
ResearchOpen Access10 Oct 2016
Scientific Reports

Volume: 6, P: 1-10

Search

Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

CRISPR-Cas3 induces broad and unidirectional genome editing in human cells

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

An iPSC-based in vitro model recapitulates human thymic epithelial development and multi-lineage specification

Optimization of the proliferation and persistency of CAR T cells derived from human induced pluripotent stem cells

Generation of hypoimmunogenic T cells from genetically engineered allogeneic human induced pluripotent stem cells

Cartilage tissue engineering identifies abnormal human induced pluripotent stem cells

Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency

Genetically engineered hypoimmunogenic cell therapy

Srf destabilizes cellular identity by suppressing cell-type-specific gene expression programs

Site-specific randomization of the endogenous genome by a regulatable CRISPR-Cas9 piggyBac system in human cells

Optimized simple culture protocol for inducing mature myotubes from MYOD1-overexpressed human iPS cells

EOS lentiviral vector selection system for human induced pluripotent stem cells

CD151 expression marks atrial- and ventricular- differentiation from human induced pluripotent stem cells

iPSC-derived type IV collagen α5-expressing kidney organoids model Alport syndrome

Establishment of quantitative and consistent in vitro skeletal muscle pathological models of myotonic dystrophy type 1 using patient-derived iPSCs

Calcium dysregulation contributes to neurodegeneration in FTLD patient iPSC-derived neurons

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