Generation of a Nonhuman Primate Model of Severe Combined Immunodeficiency Using Highly Efficient Genome Editing

Sato, Kenya; Oiwa, Ryo; Kumita, Wakako; Henry, Rachel; Sakuma, Tetsushi; Ito, Ryoji; Nozu, Ryoko; Inoue, Takashi; Katano, Ikumi; Sato, Kengo; Okahara, Norio; Okahara, Junko; Shimizu, Yoshihisa; Yamamoto, Masafumi; Hanazawa, Kisaburo; Kawakami, Takao; Kametani, Yoshie; Suzuki, Ryuji; Takahashi, Takeshi; Weinstein, Edward J.; Yamamoto, Takashi; Sakakibara, Yasubumi; Habu, Sonoko; Hata, Jun Ichi; Okano, Hideyuki; Sasaki, Erika

doi:10.1016/j.stem.2016.06.003

Cited by 143 publications

(159 citation statements)

References 52 publications

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“…In 2016, our research group focused on improving the efficiency of genome-editing technology in NHPs and reported the generation of immunodeficiency-related interleukin 2 receptor common gamma (IL2RG) KO marmosets using ZFN and TALEN [32]. In this study, we succeeded in the highly efficient generation of phenotypic individuals in the founder generation by carrying out detailed preliminary examinations using a genome editing tool.…”

Section: Target Gene Modification In Nhps Using Genome-editing Technomentioning

confidence: 99%

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Genetic engineering in nonhuman primates for human disease modeling

Sato

Sasaki

2017

J Hum Genet

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Nonhuman primate (NHP) experimental models have contributed greatly to human health research by assessing the safety and efficacy of newly developed drugs, due to their physiological and anatomical similarities to humans. To generate NHP disease models, drug-inducible methods, and surgical treatment methods have been employed. Recent developments in genetic and developmental engineering in NHPs offer new options for producing genetically modified disease models. Moreover, in recent years, genome-editing technology has emerged to further promote this trend and the generation of disease model NHPs has entered a new era. In this review, we summarize the generation of conventional disease model NHPs and discuss new solutions to the problem of mosaicism in genome-editing technology.

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Section: Target Gene Modification In Nhps Using Genome-editing Technomentioning

confidence: 99%

“…To solve this problem, it is important to verify the genome editing tools in detail prior to producing NHP models. This point has been thoroughly studied in our research [32]. First, tools for use in genome editing in NHP embryos can be easily screened by evaluating genome modification activity in cultured cells (Fig.…”

Section: Reducing Mosaicismmentioning

confidence: 99%

Genetic engineering in nonhuman primates for human disease modeling

Sato

Sasaki

2017

J Hum Genet

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“…Remarkable recent advances in genome editing technology have now made it possible to overcome these previous limitations (Sato et al 2016). Genome editing tools, i.e., engineered nucleases, bind to a target genome sequence and introduce specific double-strand breaks.…”

Section: Transgenic Techniques and Genome Editing Technology For Marmmentioning

confidence: 99%

“…Using these genome editing technologies, we recently generated X-linked SCID model marmosets by knock-out of interleukin-2 receptor subunit gamma gene (Sato et al, 2016). Currently, we are now seeking to generate marmoset models of autism spectrum disorders, including Rett syndrome (Chahrour and Zoghbi 2007;Kishi and Macklis 2005) and tuberous sclerosis complex (Ess 2010 ; Fig.…”

Section: Transgenic Techniques and Genome Editing Technology For Marmmentioning

confidence: 99%

Neuroscience Research Using Non-human Primate Models and Genome Editing

Kishi

2017

Research and Perspectives in Neurosciences

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The common marmoset (Callithrix jacchus) is a small New World non-human primate indigenous to northeastern Brazil. This species has been attracting the attention of biomedical researchers and neuroscientists for its ease of handling and colony maintenance, unique behavioral characteristics, and several human-like traits, such as enriched social vocal communication and strong relationships between parents and offspring. Its high reproductive efficiency makes it particularly amenable for use in the development of transgenic and genome editing technologies in a non-human primate model. Our group has recently generated transgenic marmosets with germ line transmission, opening new avenues in primate research.In this chapter, we describe recent advances in neuroscience and disease research using common marmosets, and we outline potential uses of genome editing in non-human primates toward the development of knock-in/knock-out marmosets.

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“…Callithrix jacchus (common marmoset; CM) is a new world monkey (7), for which genome sequence analysis and transgenic animals have been established (8,9). We reported that CM possesses CD4T cells, CD8T cells and B cells in the similar ratio in PBMC and the T cells express CD25 and various cytokines after the activation, similarly to the cells of mouse and human (8).…”

Section: Introductionmentioning

confidence: 99%