Researchers Database

Suvd BYAMBAA

    DivisionofRegenerativeMedicine,CenterformolecularMedicine Research Associate
Contact: souvdjichi.ac.jp
Last Updated :2021/10/19

Researcher Information

URL

ORCID ID

J-Global ID

Research Interests

  • Regenerative Medicine   Gene therapy   Genome-editing   

Research Areas

  • Life sciences / Molecular biology

Academic & Professional Experience

  • 2020/04  Division of Regenerative Medicine, Center for Molecular Medicine, Jichi Medical University
  • 2018/04 - 2020/03  Division of Regenerative Medicine, Center for Molecular Medicine, Jichi Medical University

Education

  • 2014/04 - 2018/04  Jichi Medical University  Division of Regenerative Medicine

Published Papers

  • Suvd Byambaa, Hideki Uosaki, Tsukasa Ohmori, Hiromasa Hara, Hitoshi Endo, Osamu Nureki, Yutaka Hanazono
    Molecular therapy. Methods & clinical development 20 451 - 462 2021/03 
    We conducted two lines of genome-editing experiments of mouse hematopoietic stem cells (HSCs) with the clustered regularly interspaced short palindromic repeat (CRISPR) and CRISPR-associated protein 9 (Cas9). First, to evaluate the genome-editing efficiency in mouse bona fide HSCs, we knocked out integrin alpha 2b (Itga2b) with Cas9 ribonucleoprotein (Cas9/RNP) and performed serial transplantation in mice. The knockout efficiency was estimated at approximately 15%. Second, giving an example of X-linked severe combined immunodeficiency (X-SCID) as a target genetic disease, we showed a proof-of-concept of universal gene correction, allowing rescue of most of X-SCID mutations, in a completely non-viral setting. We inserted partial cDNA of interleukin-2 receptor gamma chain (Il2rg) into intron 1 of Il2rg via non-homologous end-joining (NHEJ) with Cas9/RNP and a homology-independent targeted integration (HITI)-based construct. Repaired HSCs reconstituted T lymphocytes and thymuses in SCID mice. Our results show that a non-viral genome-editing of HSCs with CRISPR/Cas9 will help cure genetic diseases.
  • Suvd Byambaa, Hideki Uosaki, Hiromasa Hara, Yasumitsu Nagao, Tomoyuki Abe, Hiroaki Shibata, Osamu Nureki, Tsukasa Ohmori, Yutaka Hanazono
    Experimental animals 69 (2) 189 - 198 2020/04 [Refereed][Not invited]
     
    X-linked severe combined immunodeficiency (X-SCID) is an inherited genetic disorder. A majority of X-SCID subjects carries point mutations in the Interleukin-2 receptor gamma chain (IL2RG) gene. In contrast, Il2rg-knockout mice recapitulating X-SCID phenotype lack a large part of Il2rg instead of point mutations. In this study, we generated novel X-SCID mouse strains with small insertion and deletion (InDel) mutations in Il2rg by using clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9. To this end, we injected Streptococcus pyogenes Cas9 (SpCas9) mRNA and single guide RNA targeting the exon 2, 3 or 4 of Il2rg into mouse zygotes. In the F0 generation, we obtained 35 pups and 25 out of them were positive for Surveyor assay, and most of mutants displayed dramatic reductions of T and B lymphocytes in the peripheral blood. By amplicon sequencing, 15 out of 31 founder mice were determined as monoallelic mutants with possible minor mosaicisms while 10 mice were mosaic. Finally, we established new strains with 7-nucleotide deletion and 1-nucleotide insertions in the exon 2 and the exons 3 and 4, respectively. Although no IL2RG protein was detected on T cells of exons 3 and 4 mutants, IL2RG protein was unexpectedly detected in the exon 2 mutants. These data indicated that CRISPR/Cas9 targeting Il2rg causes InDel mutations effectively and generates genetically X-SCID mice. Genetic mutations, however, did not necessarily grant phenotypical alteration, which requires an intensive analysis after establishing a strain to confirm their phenotypes.

Conference Activities & Talks

  • Phenotypical Correction of X-SCID Mice After CRISPR/Cas9-mediated UNIVERSAL Genome-Editing Therapy of Hematopoietic Stem Cells
    Suvd Byambaa, Hideki Uosaki, Hiromasa Hara, Hiroaki Shibata, Tomoyuki Abe, Yasumitsu Nagao, Osamu Nureki, Tsukasa Ohmori, Yutaka Hanazono
    Frontiers in Genome Engineering  2019/11
  • Phenotypical Correction of X-SCID Mice After CRISPR/Cas9-mediated Genome-Editing Therapy of Hematopoietic Stem Cells
    Suvd Byambaa, Hideki Uosaki, Hiromasa Hara, Hiroaki Shibata, Tomoyuki Abe, Yasumitsu Nagao, Osamu Nureki, Tsukasa Ohmori, Yutaka Hanazono
    JSGCT  2019/07
  • Genome Editing of Murine Hematopoietic Stem Cells by Cas9/RNP Targeting Integrin-alpha IIb Gene
    Suvd BYAMBAA, Hideki UOSAKI, Tsukasa OHMORI, Yutaka HANAZONO
    JSGE  2019/06
  • Cure of X-SCID Mice After Ex Vivo Non-viral Genome-Editing Therapy of Hematopoietic Stem Cells
    Suvd Byambaa, Hideki Uosaki, Hiromasa Hara, Hiroaki Shibata, Tomoyuki Abe, Hiroko Hayakawa, Yasumitsu Nagao, Osamu Nureki, Tsukasa Ohmori, Yutaka Hanazono
    ASGCT  2019/05
  • Targeted Gene Integration in Hematopoietic Stem and Progenitor Cells with Cas9-RNP Method to Universally Correct X-SCID Mutations
    Byambaa S, Uosaki H, Hara H, Shibata H, Abe T, Nagao Y, Nureki O, Ohmori T, Hanazono Y
    第41回日本分子生物学会年会 2018年11月30日  2018/11
  • Targeted Genome Editing of Murine Hematopoietic Stem Cells by CRISPR/Cas9
    BYAMBAA S, UOSAKI H, HARA H, SHIBATA H, ABE T, NAGAO Y, NUREKI O, OHMORI T, HANAZONO Y
    日本遺伝子細胞治療学会学術集会  2018/07
  • Novel SCID mice generation by CRISPR/Cas9
    BYAMBAA S, UOSAKI H, HARA H, SHIBATA H, ABE T, NAGAO Y, NUREKI O, OHMORI T, HANAZONO Y
    第65回日本実験動物学会  2018/05
  • CRISPR/Cas9-Mediated Targeted Genome Editing of Hematopoietic Stem and Progenitor Cells
    Suvd BYAMBAA, Hideki UOSAKI, Hiromasa HARA, Hiroaki SHIBATA, Tomoyuki ABE, Yasumitsu NAGAO, Osamu NUREKI, Tsukasa OHMORI, Yutaka HANAZONO
    Takeda Symposium  2018/02
  • Resistance of Mouse Hematopoietic Stem and Progenitor Cells to the Genome-Editing with Cas9
    Byambaa S, Uosaki H, Hara H, Abe T, Nagao Y, Nureki O, Ohmori T, Hanazono Y
    ASGCT  2017/05

Awards & Honors

  • 2019/11 Frontiers in Genome Engineering Young Scientist Award
  • 2019/05 ASGCT Meritorious Abstract Travel Awards


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