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From Genomics to Gene Therapy: Induced Pluripotent Stem Cells Meet Genome Editing

Annual review of genetics, 2015-11, Vol.49 (1), p.47-70

2015

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Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

Journal of controlled release, 2022-02, Vol.342, p.345-361

2022

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

Nature communications, 2021-12, Vol.12 (1), p.7101-13, Article 7101

2021

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

Nature communications, 2020-03, Vol.11 (1), p.1334-18, Article 1334

2020

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

Nature communications, 2019-12, Vol.10 (1), p.5302-13, Article 5302

2019

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Hair Follicle Dermal Stem Cells Regenerate the Dermal Sheath, Repopulate the Dermal Papilla, and Modulate Hair Type

Developmental cell, 2014-12, Vol.31 (5), p.543-558

2014

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PKD1 -Dependent Renal Cystogenesis in Human Induced Pluripotent Stem Cell-Derived Ureteric Bud/Collecting Duct Organoids

Journal of the American Society of Nephrology, 2020-10, Vol.31 (10), p.2355-2371

2020

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Evolutionarily Distinctive Transcriptional and Signaling Programs Drive Human Germ Cell Lineage Specification from Pluripotent Stem Cells

Cell stem cell, 2017-10, Vol.21 (4), p.517-532.e5

2017

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Stage-Specific Optimization of Activin/Nodal and BMP Signaling Promotes Cardiac Differentiation of Mouse and Human Pluripotent Stem Cell Lines

Cell stem cell, 2011-02, Vol.8 (2), p.228-240

2011

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Efficient genomic correction methods in human iPS cells using CRISPR–Cas9 system

Methods (San Diego, Calif.), 2016-05, Vol.101, p.27-35

2016

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Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9

Stem cell reports, 2015-01, Vol.4 (1), p.143-154

2015

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Genome Editing Gene Therapy for Duchenne Muscular Dystrophy

Journal of neuromuscular diseases, 2015-09, Vol.2 (4), p.343-355

Hotta, Akitsu

2015

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Isolation of MECP2-nuLl Rett Syndrome patient hiPS cells and isogenic controls through X-chromosome inactivation

Human molecular genetics, 2011-06, Vol.20 (11), p.2103-2115

2011

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Dual inhibition of TMPRSS2 and Cathepsin Bprevents SARS-CoV-2 infection in iPS cells

Molecular therapy. Nucleic acids, 2021-12, Vol.26, p.1107-1114

2021

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Efficient and reproducible myogenic differentiation from human iPS cells: prospects for modeling Miyoshi Myopathy in vitro

PloS one, 2013-04, Vol.8 (4), p.e61540-e61540

2013

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

Nature biomedical engineering, 2021-05, Vol.5 (5), p.429-440

2021

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Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases

International Journal of Molecular Sciences, 2015-10, Vol.16 (10), p.24751-24771

2015

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Direct Comparison of Autologous and Allogeneic Transplantation of iPSC-Derived Neural Cells in the Brain of a Nonhuman Primate

Stem cell reports, 2013-10, Vol.1 (4), p.283-292

2013

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

Scientific reports, 2023-01, Vol.13 (1), p.94-94, Article 94

2023

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Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model

Cell reports (Cambridge), 2016-06, Vol.15 (10), p.2301-2312

2016

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Genetic correction using engineered nucleases for gene therapy applications

Development, growth & differentiation, 2014-01, Vol.56 (1), p.63-77

2014

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Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy

Stem cells international, 2017-01, Vol.2017 (2017), p.1-11

2017

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Constitutive heterochromatin reorganization during somatic cell reprogramming

The EMBO journal, 2011-05, Vol.30 (9), p.1778-1789

2011

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iMSC-mediated delivery of ACVR2B-Fc fusion protein reduces heterotopic ossification in a mouse model of fibrodysplasia ossificans progressiva

Stem cell research & therapy, 2024-03, Vol.15 (1), p.83-83, Article 83

2024

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From Genomics to Gene Therapy: Induced Pluripotent Stem Cells Meet Genome Editing

Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

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

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

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

Hair Follicle Dermal Stem Cells Regenerate the Dermal Sheath, Repopulate the Dermal Papilla, and Modulate Hair Type

PKD1 -Dependent Renal Cystogenesis in Human Induced Pluripotent Stem Cell-Derived Ureteric Bud/Collecting Duct Organoids

Evolutionarily Distinctive Transcriptional and Signaling Programs Drive Human Germ Cell Lineage Specification from Pluripotent Stem Cells

Stage-Specific Optimization of Activin/Nodal and BMP Signaling Promotes Cardiac Differentiation of Mouse and Human Pluripotent Stem Cell Lines

The Src/c-Abl pathway is a potential therapeutic target in amyotrophic lateral sclerosis

Efficient genomic correction methods in human iPS cells using CRISPR–Cas9 system

Precise correction of the dystrophin gene in duchenne muscular dystrophy patient induced pluripotent stem cells by TALEN and CRISPR-Cas9

Genome Editing Gene Therapy for Duchenne Muscular Dystrophy

Isolation of MECP2-nuLl Rett Syndrome patient hiPS cells and isogenic controls through X-chromosome inactivation

Dual inhibition of TMPRSS2 and Cathepsin Bprevents SARS-CoV-2 infection in iPS cells

Efficient and reproducible myogenic differentiation from human iPS cells: prospects for modeling Miyoshi Myopathy in vitro

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

Minimizing off-Target Mutagenesis Risks Caused by Programmable Nucleases

Direct Comparison of Autologous and Allogeneic Transplantation of iPSC-Derived Neural Cells in the Brain of a Nonhuman Primate

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

Concordant but Varied Phenotypes among Duchenne Muscular Dystrophy Patient-Specific Myoblasts Derived using a Human iPSC-Based Model

Genetic correction using engineered nucleases for gene therapy applications

Cellular Reprogramming, Genome Editing, and Alternative CRISPR Cas9 Technologies for Precise Gene Therapy of Duchenne Muscular Dystrophy

Constitutive heterochromatin reorganization during somatic cell reprogramming

iMSC-mediated delivery of ACVR2B-Fc fusion protein reduces heterotopic ossification in a mouse model of fibrodysplasia ossificans progressiva

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