Laurenti, E. & Gottgens, B. From haematopoietic stem cells to advanced differentiation landscapes. Nature 553, 418–426 (2018).
Bauer, T. R. Jr. et al. Correction of the illness phenotype in canine leukocyte adhesion deficiency utilizing ex vivo hematopoietic stem cell gene remedy. Blood 108, 3313–3320 (2006).
Blaese, R. M. et al. T lymphocyte-directed gene remedy for ADA-SCID: preliminary trial outcomes after 4 years. Science 270, 475–480 (1995).
Boztug, Okay. et al. Stem-cell gene remedy for the Wiskott-Aldrich syndrome. N. Engl. J. Med. 363, 1918–1927 (2010).
Cowan, M. J. et al. Early final result of a section I/II scientific trial (NCT03538899) of gene-corrected autologous CD34+ hematopoietic cells and low-exposure busulfan in newly identified sufferers with Artemis-deficient extreme mixed immunodeficiency (ART-SCID). Biol. Blood Marrow Transpl. 26, S88–S89 (2020).
Gaspar, H. B. et al. Gene remedy of X-linked extreme mixed immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364, 2181–2187 (2004).
Kanter, J. et al. Biologic and scientific efficacy of LentiGlobin for sickle cell illness. N. Engl. J. Med. 386, 617–628 (2022).
Kohn, L. A. & Kohn, D. B. Gene therapies for main immune deficiencies. Entrance. Immunol. 12, 648951 (2021).
Kondo, M. et al. Biology of hematopoietic stem cells and progenitors: implications for scientific software. Annu Rev. Immunol. 21, 759–806 (2003).
Locatelli, F. et al. Betibeglogene autotemcel gene remedy for non-β0/β0 genotype β-thalassemia. N. Engl. J. Med. 386, 415–427 (2022).
Malech, H. L. et al. Extended manufacturing of NADPH oxidase-corrected granulocytes after gene remedy of power granulomatous illness. Proc. Natl Acad. Sci. USA 94, 12133–12138 (1997).
Morgan, R. A., Grey, D., Lomova, A. & Kohn, D. B. Hematopoietic stem cell gene remedy: progress and classes realized. Cell Stem Cell 21, 574–590 (2017).
Sago, C. D. et al. Nanoparticles that ship RNA to bone marrow recognized by in vivo directed evolution. J. Am. Chem. Soc. 140, 17095–17105 (2018).
Shi, D., Toyonaga, S. & Anderson, D. G. In vivo RNA supply to hematopoietic stem and progenitor cells by way of focused lipid nanoparticles. Nano Lett. 23, 2938–2944 (2023).
Sou, Okay., Goins, B., Oyajobi, B. O., Travi, B. L. & Phillips, W. T. Bone marrow-targeted liposomal carriers. Skilled Opin. Drug Deliv. 8, 317–328 (2011).
Sou, Okay., Klipper, R., Goins, B., Tsuchida, E. & Phillips, W. T. Circulation kinetics and organ distribution of Hb-vesicles developed as a purple blood cell substitute. J. Pharmacol. Exp. Ther. 312, 702–709 (2005).
Xue, L. et al. Rational design of bisphosphonate lipid-like supplies for mRNA supply to the bone microenvironment. J. Am. Chem. Soc. 144, 9926–9937 (2022).
Boulais, P. E. & Frenette, P. S. Making sense of hematopoietic stem cell niches. Blood 125, 2621–2629 (2015).
Ikonomi, N., Kuhlwein, S. D., Schwab, J. D. & Kestler, H. A. Awakening the HSC: dynamic modeling of HSC upkeep unravels regulation of the TP53 pathway and quiescence. Entrance. Physiol. 11, 848 (2020).
Li, J. Quiescence regulators for hematopoietic stem cell. Exp. Hematol. 39, 511–520 (2011).
Man, Y., Yao, X., Yang, T. & Wang, Y. Hematopoietic stem cell area of interest throughout homeostasis, malignancy, and bone marrow transplantation. Entrance. Cell Dev. Biol. 9, 621214 (2021).
Nakamura-Ishizu, A., Takizawa, H. & Suda, T. The evaluation, roles and regulation of quiescence in hematopoietic stem cells. Growth 141, 4656–4666 (2014).
Eppert, Okay. et al. Stem cell gene expression applications affect scientific final result in human leukemia. Nat. Med. 17, 1086–1093 (2011).
Lapidot, T. et al. A cell initiating human acute myeloid leukaemia after transplantation into SCID mice. Nature 367, 645–648 (1994).
Mandal, T., Beck, M., Kirsten, N., Linden, M. & Buske, C. Concentrating on murine leukemic stem cells by antibody functionalized mesoporous silica nanoparticles. Sci. Rep. 8, 989 (2018).
Pei, S. & Jordan, C. T. How shut are we to concentrating on the leukemia stem cell? Finest Pract. Res. Clin. Haematol. 25, 415–418 (2012).
Li, C. et al. Prophylactic in vivo hematopoietic stem cell gene remedy with an immune checkpoint inhibitor reverses tumor development in syngeneic mouse tumor fashions. Most cancers Res. 80, 549–560 (2020).
Li, C. et al. In vivo HSPC gene remedy with base editors permits for environment friendly reactivation of fetal globin in beta-YAC mice. Blood Adv. 5, 1122–1135 (2021).
Li, C. et al. In vivo HSC gene remedy utilizing a bi-modular HDAd5/35++ vector cures sickle cell illness in a mouse mannequin. Mol. Ther. 29, 822–837 (2021).
Li, C. et al. Protected and environment friendly in vivo hematopoietic stem cell transduction in nonhuman primates utilizing HDAd5/35++ vectors. Mol. Ther. Strategies Clin. Dev. 24, 127–141 (2022).
Psatha, N. et al. Enhanced HbF reactivation by multiplex mutagenesis of thalassemic CD34+ cells in vitro and in vivo. Blood 138, 1540–1553 (2021).
Muruve, D. A., Barnes, M. J., Stillman, I. E. & Libermann, T. A. Adenoviral gene remedy results in fast induction of a number of chemokines and acute neutrophil-dependent hepatic damage in vivo. Hum. Gene Ther. 10, 965–976 (1999).
Sweeney, C. L. & De Ravin, S. S. The promise of in vivo HSC prime modifying. Blood 141, 2039–2040 (2023).
Worgall, S., Wolff, G., Falck-Pedersen, E. & Crystal, R. G. Innate immune mechanisms dominate elimination of adenoviral vectors following in vivo administration. Hum. Gene Ther. 8, 37–44 (1997).
Lek, A. et al. Dying after high-dose rAAV9 gene remedy in a affected person with Duchenne’s muscular dystrophy. N. Engl. J. Med. 389, 1203–1210 (2023).
Hou, X., Zaks, T., Langer, R. & Dong, Y. Lipid nanoparticles for mRNA supply. Nat. Rev. Mater. 6, 1078–1094 (2021).
Cheng, Q. et al. Selective organ concentrating on (SORT) nanoparticles for tissue-specific mRNA supply and CRISPR-Cas gene modifying. Nat. Nanotechnol. 15, 313–320 (2020).
Dilliard, S. A., Cheng, Q. & Siegwart, D. J. On the mechanism of tissue-specific mRNA supply by selective organ concentrating on nanoparticles. Proc. Natl Acad. Sci. USA 118, e2109256118 (2021).
Dilliard, S. A. & Siegwart, D. J. Passive, lively and endogenous organ-targeted lipid and polymer nanoparticles for supply of genetic medicine. Nat. Rev. Mater. 8, 282–300 (2023).
Farbiak, L. et al. All-in-one dendrimer-based lipid nanoparticles allow exact HDR-mediated gene modifying in vivo. Adv. Mater. 33, e2006619 (2021).
Liu, S. et al. Membrane-destabilizing ionizable phospholipids for organ-selective mRNA supply and CRISPR-Cas gene modifying. Nat. Mater. 20, 701–710 (2021).
Liu, S. et al. Zwitterionic phospholipidation of cationic polymers facilitates systemic mRNA supply to spleen and lymph nodes. J. Am. Chem. Soc. 143, 21321–21330 (2021).
Wang, X. et al. Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs) utilizing a number of technical strategies for tissue-specific mRNA supply. Nat. Protoc. 18, 265–291 (2023).
Wei, T., Cheng, Q., Min, Y. L., Olson, E. N. & Siegwart, D. J. Systemic nanoparticle supply of CRISPR-Cas9 ribonucleoproteins for efficient tissue particular genome modifying. Nat. Commun. 11, 3232 (2020).
Zhang, D. et al. Enhancing CRISPR/Cas gene modifying by modulating mobile mechanical properties for most cancers remedy. Nat. Nanotechnol. 17, 777–787 (2022).
Wu, L. C. et al. Correction of sickle cell illness by homologous recombination in embryonic stem cells. Blood 108, 1183–1188 (2006).
Metais, J. Y. et al. Genome modifying of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv. 3, 3379–3392 (2019).
Newby, G. A. et al. Base modifying of haematopoietic stem cells rescues sickle cell illness in mice. Nature 595, 295–302 (2021).
Stavropoulou, V., Peters, A. & Schwaller, J. Aggressive leukemia pushed by MLL-AF9. Mol. Cell Oncol. 5, e1241854 (2018).
Hou, X. et al. Vitamin lipid nanoparticles allow adoptive macrophage switch for the remedy of multidrug-resistant bacterial sepsis. Nat. Nanotechnol. 15, 41–46 (2020).
Morales-Tenorio, M. et al. Potential pharmacological methods concentrating on the Niemann-Decide C1 receptor and Ebola virus glycoprotein interplay. Eur. J. Med. Chem. 223, 113654 (2021).
Zuo, Y. et al. Managed supply of a neurotransmitter-agonist conjugate for practical restoration after extreme spinal twine damage. Nat. Nanotechnol. 18, 1230–1240 (2023).
Boike, L., Henning, N. J. & Nomura, D. Okay. Advances in covalent drug discovery. Nat. Rev. Drug Discov. 21, 881–898 (2022).
Zhou, Okay. et al. Modular degradable dendrimers allow small RNAs to increase survival in an aggressive liver most cancers mannequin. Proc. Natl Acad. Sci. USA 113, 520–525 (2016).
Madisen, L. et al. A sturdy and high-throughput Cre reporting and characterization system for the entire mouse mind. Nat. Neurosci. 13, 133–140 (2010).
Akinc, A. et al. Focused supply of RNAi therapeutics with endogenous and exogenous ligand-based mechanisms. Mol. Ther. 18, 1357–1364 (2010).
Kim, M. et al. Engineered ionizable lipid nanoparticles for focused supply of RNA therapeutics into various kinds of cells within the liver. Sci. Adv. 7, eabf4398 (2021).
Enache, O. M. et al. Cas9 prompts the p53 pathway and selects for p53-inactivating mutations. Nat. Genet. 52, 662–668 (2020).
Haapaniemi, E., Botla, S., Persson, J., Schmierer, B. & Taipale, J. CRISPR-Cas9 genome modifying induces a p53-mediated DNA harm response. Nat. Med. 24, 927–930 (2018).
Leibowitz, M. L. et al. Chromothripsis as an on-target consequence of CRISPR-Cas9 genome modifying. Nat. Genet. 53, 895–905 (2021).
Mayuranathan, T. et al. Potent and uniform fetal hemoglobin induction by way of base modifying. Nat. Genet. 55, 1210–1220 (2023).
Zuccaro, M. V. et al. Allele-specific chromosome removing after Cas9 cleavage in human embryos. Cell 183, 1650–1664e1615 (2020).
Anzalone, A. V. et al. Search-and-replace genome modifying with out double-strand breaks or donor DNA. Nature 576, 149–157 (2019).
Miller, S. M. et al. Steady evolution of SpCas9 variants suitable with non-G PAMs. Nat. Biotechnol. 38, 471–481 (2020).
Richter, M. F. et al. Phage-assisted evolution of an adenine base editor with improved Cas area compatibility and exercise. Nat. Biotechnol. 38, 883–891 (2020).
Breda, L. et al. In vivo hematopoietic stem cell modification by mRNA supply. Science 381, 436–443 (2023).
Marschalek, R. MLL leukemia and future remedy methods. Arch. Pharm. 348, 221–228 (2015).
Stavropoulou, V. et al. MLL-AF9 expression in hematopoietic stem cells drives a extremely invasive AML expressing EMT-related genes linked to poor final result. Most cancers Cell 30, 43–58 (2016).
Kang, X. et al. The ITIM-containing receptor LAIR1 is crucial for acute myeloid leukaemia improvement. Nat. Cell Biol. 17, 665–677 (2015).
Wu, G. et al. LILRB3 helps acute myeloid leukemia improvement and regulates T-cell antitumor immune responses by the TRAF2–cFLIP–NF-κB signaling axis. Nat. Most cancers 2, 1170–1184 (2021).
Zheng, J. et al. Inhibitory receptors bind ANGPTLs and assist blood stem cells and leukaemia improvement. Nature 485, 656–660 (2012).
Itskovich, S. S. et al. MBNL1 regulates important various RNA splicing patterns in MLL-rearranged leukemia. Nat. Commun. 11, 2369 (2020).
Barreto, I. V. et al. Leukemic stem cell: a mini-review on scientific views. Entrance. Oncol. 12, 931050 (2022).
Clement, Okay. et al. CRISPResso2 offers correct and fast genome modifying sequence evaluation. Nat. Biotechnol. 37, 224–226 (2019).