Yang Liu

About

I am a translational researcher working at the intersection of hematopoietic stem cell biology, gene therapy, and regenerative medicine.

My work focuses on developing hematopoietic stem cell gene therapies for genetic blood disorders and next-generation immunotherapies, using AAV capsid engineering and CRISPR-Cas9 genome editing. Originally trained in orthopaedic and musculoskeletal research, I bring a tissue-regeneration perspective to stem cell therapy and clinical translation.

Across these areas, I am driven by a simple question: how can we turn strong laboratory science into safe, effective, and accessible therapies for patients?

我是一名关注临床转化的研究者，工作在造血干细胞生物学、基因治疗和再生医学的交叉地带。

现在，我主要研究如何把造血干细胞变成更可靠的治疗平台，用于遗传性血液疾病和下一代免疫治疗。 具体来说，我结合 AAV 衣壳工程和 CRISPR-Cas9 基因组编辑，探索如何更精准、更安全地把治疗性基因和编辑工具送进目标细胞，并一步步推向真正可用的临床方案。我的科研起点来自骨科与肌肉骨骼再生研究。博士期间，我研究过肌腱来源干细胞、骨修复，以及促进肌腱愈合的小分子策略。那段经历让我一直相信，干细胞研究不只是理解细胞如何工作，更重要的是思考它们如何帮助受损组织恢复功能，最终改善患者的生活。

贯穿这些研究的，是一个始终吸引我的问题： 怎样把有说服力的实验室发现，变成安全、有效、并且真正能到达患者身边的治疗方法？

Karolinska ATMP Center · Center for Hematology and Regenerative Medicine, MedH, Karolinska Institutet

1,292 Citations · till 2026
21 h-index
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Research Interests

01 Hematology & Gene Therapy

Developing and translating gene therapies for inherited bone marrow failure syndromes, with a focus on novel genetic engineering strategies on hematopoietic stem cells.

  • Co-developed a clinically applicable lentiviral vector for RPS19-deficient DBA, supporting an ongoing clinical trial
  • Contributed to FDA Orphan Drug Designation for the DBA gene therapy program
  • Built a humanized DBA disease model using Cas9-mediated HDR via AAV
  • Established the novel HEAL directed-evolution platform for safe, efficient in vivo HSC genetic engineering
HSC genetic engineeringDirected evolution & AAVCRISPR/Cas9-mediated HDRStem cell biologyATMPClinical translation
02 Musculoskeletal & Regenerative Medicine

Earlier and continuing work on tendon-derived stem cells, tendon and bone healing, and small-molecule and ncRNA strategies for musculoskeletal regeneration.

  • Co-inventor on a small-molecule patent for enhancing tendon injury healing
  • Characterized dysfunction of CFTR in tenogenic differentiation and tendon repair
  • Studied the roles of lncRNA and/or miRNA in tendon healing
  • Top-10 most cited papers in the Journal of Orthopaedic Translation
Tendon-derived stem cellsBone regenerationSmall moleculesncRNAMSCs

Funding Support

Åke Wiberg FoundationCIMED · Region StockholmStiftelsen Lars Hiertas MinneRoyal Physiographic Society of LundSwedish Children Cancer FoundationWenner-Gren FoundationKarolinska InstitutetThe Chinese University of Hong Kong

Publications

血液学与基因治疗 Hematology & Gene Therapy

  • Neonatal exchange blood is a rich source of hematopoietic stem and progenitor cells for transplantation.
    Liu Y, Giusti P, Qian H, Wu PC, Wang T, De Haan S, Toporski J, Wikman A, Höglund P, Deschmann E, Fiskesund R. · Haematologica, 2026
    PubMed →
  • Perspectives of current understanding and therapeutics of Diamond-Blackfan anemia.
    Liu Y, Karlsson S. · Leukemia 38(1):1-9, 2024 (co-corresponding)
    PubMed →
  • Engineered human Diamond-Blackfan anemia disease model confirms therapeutic effects of clinically applicable lentiviral vector at single-cell resolution.
    Liu Y, Schmiderer L, Hjort M, Lang S, Bremborg T, Rydström A, Schambach A, Larsson J, Karlsson S. · Haematologica 108(11):3095-3109, 2023 (co-corresponding)
    PubMed →
  • Successful gene therapy of Diamond-Blackfan anemia in a mouse model and human CD34+ cord blood hematopoietic stem cells using a clinically applicable lentiviral vector.
    Liu Y, Dahl M, Debnath S, Rothe M, Smith EM, Grahn THM, Warsi S, Chen J, Flygare J, Schambach A, Karlsson S. · Haematologica 107(2):446-456, 2022 (co-corresponding)
    PubMed →
  • Schlafen2 is a regulator of quiescence in adult murine hematopoietic stem cells.
    Warsi S, Dahl M, Smith EMK, Rydstrom A, Mansell E, Sigurdsson V, Sjoberg J, Soneji S, Rorby E, Siva K, Grahn THM, Liu Y, Blank U, Karlsson G, Karlsson S. · Haematologica 107(12):2884-2896, 2022
    PubMed →
  • Bone marrow transplantation without myeloablative conditioning in a mouse model for Diamond-Blackfan anemia corrects the disease phenotype.
    Dahl M, Warsi S, Liu Y, Debnath S, Billing M, Siva K, Flygare J, Karlsson S. · Exp Hematol 99:44-53.e2, 2021
    PubMed →
  • S100A6 is a critical regulator of hematopoietic stem cells.
    Grahn THM, Niroula A, Végvári Á, Oburoglu L, Pertesi M, Warsi S, Safi F, Miharada N, Garcia SC, Siva K, Liu Y, Rörby E, Nilsson B, Zubarev RA, Karlsson S. · Leukemia 34(12):3323-3337, 2020
    PubMed →

肌肉骨骼与再生医学 Musculoskeletal & Regenerative Medicine

  • Vasoactive Intestinal Peptide stimulates BM-MSC osteogenesis via Wnt/β-catenin and promotes rat skull defect repair.
    Shi L, Feng L, Zhu ML, Yang ZM, Wu TY, Xu J, Liu Y, Lin WP, Lo JHT, Zhang JF, Li G. · Stem Cells Dev 29(10):655-666, 2020
    PubMed →
  • MiR-378a suppresses tenogenic differentiation and tendon repair by targeting at TGF-β2.
    Liu Y, Feng L, Xu J, Yang Z, Wu T, Zhang J, Shi L, Zhu D, Zhang J, Li G. · Stem Cell Res Ther 10(1):108, 2019
    PubMed →
  • Identification of an anti-inflammation protein, Annexin A1, in tendon-derived stem cells of cystic fibrosis mice: a comparative proteomic analysis.
    Liu Y, Feng L, Wang H, Wang YJ, Chan HC, Jiang XH, Fu WM, Li G, Zhang JF. · Proteomics Clin Appl 12(6):e1700162, 2018
    PubMed →
  • Enhancement of bone regeneration with the accordion technique via HIF-1α/VEGF activation in a rat distraction osteogenesis model.
    Xu J, Sun Y, Wu T, Liu Y, Shi L, Zhang J, Kang Q, Chai Y, Li G. · J Tissue Eng Regen Med 12(2):e1268-e1276, 2018
    PubMed →
  • The role of long non-coding RNA H19 in musculoskeletal system: a new player in an old game.
    Liu Y, Li G, Zhang JF. · Exp Cell Res 360(2):61-65, 2017
    PubMed →
  • CFTR mediates tenogenic differentiation of tendon-derived stem cells and tendon repair.
    Liu Y, Xu J, Xu L, Wu T, Sun Y, Lee YW, Wang B, Chan HC, Jiang X, Zhang J, Li G. · FASEB J 31(9):3800-3815, 2017
    PubMed →
  • Current concepts on tenogenic differentiation and clinical applications.
    Liu Y, Suen CW, Zhang JF, Li G. · J Orthop Translat 9:28-42, 2017 (Top-10 most cited)
    PubMed →
  • Long noncoding RNA H19 accelerates tenogenic differentiation and promotes tendon healing through targeting miR-29b-3p and activating TGF-β1 signaling.
    Lu YF, Liu Y, Fu WM, Xu J, Wang B, Sun YX, Wu TY, Xu LL, Chan KM, Zhang JF, Li G. · FASEB J 31(3):954-964, 2017 (co-first)
    PubMed →
  • Effects of Sclerostin Antibody on the healing of femoral fractures in ovariectomised rats.
    Liu Y, Rui Y, Cheng TY, Huang S, Xu L, Meng F, Lee WY, Zhang T, Li N, Li C, Ke H, Li G. · Calcif Tissue Int 98(3):263-74, 2016
    PubMed →
  • The use of cocultured mesenchymal stem cells with tendon-derived stem cells as a better cell source for tendon repair.
    Wu T, Liu Y, Wang B, Sun Y, Xu J, Yuk-Wai LW, Xu L, Zhang J, Li G. · Tissue Eng Part A 22(19-20):1229-1240, 2016
    PubMed →
  • Human fetal mesenchymal stem cell secretome enhances bone consolidation in distraction osteogenesis.
    Xu J, Wang B, Sun Y, Wu T, Liu Y, Zhang J, Lee WY, Pan X, Chai Y, Li G. · Stem Cell Res Ther 7(1):134, 2016
    PubMed →
  • Small nuclear ribonucleoprotein polypeptide N (Sm51) promotes osteogenic differentiation of BM-MSCs by regulating Runx2.
    Meng F, Xu L, Huang S, Liu Y, Hou Y, Wang K, Jiang X, Li G. · Cell Tissue Res 366(1):155-62, 2016
    PubMed →
  • U0126 promotes osteogenesis of rat bone-marrow-derived mesenchymal stem cells by activating BMP/Smad signaling pathway.
    Xu L, Liu Y, Hou Y, Wang K, Wong Y, Lin S, Li G. · Cell Tissue Res 359(2):537-545, 2015 (co-first)
    PubMed →
  • Sox11-modified MSCs accelerate bone fracture healing.
    Xu L, Huang S, Hou Y, Liu Y, Ni M, Meng F, Wang K, Rui Y, Jiang X, Li G. · FASEB J 29(4):1143-52, 2015
    PubMed →
  • Epigenetic memory gained by priming with osteogenic induction medium improves osteogenesis and other properties of MSCs.
    Rui Y, Xu L, Chen R, Zhang T, Lin S, Hou Y, Liu Y, Meng F, Liu Z, Ni M, Tsang KS, Yang F, Wang C, Chan HC, Jiang X, Li G. · Sci Rep 5:11056, 2015
    PubMed →
  • miR-21 overexpressing MSCs accelerate fracture healing in a rat closed femur fracture model.
    Sun Y, Xu L, Huang S, Hou Y, Liu Y, Chan KM, Pan XH, Li G. · Biomed Res Int 2015:412327, 2015
    PubMed →
  • The roles of mesenchymal stem cells in tissue repair and disease modification.
    Wu T, Liu Y, Wang B, Li G. · Curr Stem Cell Res Ther 9(5):424-31, 2014
    PubMed →
  • Engineered scaffold-free tendon tissue produced by tendon-derived stem cells.
    Ni M, Rui YF, Tan Q, Liu Y, Xu LL, Chan KM, Wang Y, Li G. · Biomaterials 34(8):2024-37, 2013
    PubMed →

Contact

Email: younghope11@gmail.com
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