Laurenti, E. & Gottgens, B. From haematopoietic stem cells to complex differentiation landscapes. Nature 553, 418426 (2018).
Article CAS PubMed PubMed Central Google Scholar
Bauer, T. R. Jr. et al. Correction of the disease phenotype in canine leukocyte adhesion deficiency using ex vivo hematopoietic stem cell gene therapy. Blood 108, 33133320 (2006).
Article CAS PubMed PubMed Central Google Scholar
Blaese, R. M. et al. T lymphocyte-directed gene therapy for ADA-SCID: initial trial results after 4years. Science 270, 475480 (1995).
Article CAS PubMed Google Scholar
Boztug, K. et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. N. Engl. J. Med. 363, 19181927 (2010).
Article CAS PubMed PubMed Central Google Scholar
Cowan, M. J. et al. Early outcome of a phase I/II clinical trial (NCT03538899) of gene-corrected autologous CD34+ hematopoietic cells and low-exposure busulfan in newly diagnosed patients with Artemis-deficient severe combined immunodeficiency (ART-SCID). Biol. Blood Marrow Transpl. 26, S88S89 (2020).
Article Google Scholar
Gaspar, H. B. et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364, 21812187 (2004).
Article CAS PubMed Google Scholar
Kanter, J. et al. Biologic and clinical efficacy of LentiGlobin for sickle cell disease. N. Engl. J. Med. 386, 617628 (2022).
Article CAS PubMed Google Scholar
Kohn, L. A. & Kohn, D. B. Gene therapies for primary immune deficiencies. Front. Immunol. 12, 648951 (2021).
Article CAS PubMed PubMed Central Google Scholar
Kondo, M. et al. Biology of hematopoietic stem cells and progenitors: implications for clinical application. Annu Rev. Immunol. 21, 759806 (2003).
Article CAS PubMed Google Scholar
Locatelli, F. et al. Betibeglogene autotemcel gene therapy for non-0/0 genotype -thalassemia. N. Engl. J. Med. 386, 415427 (2022).
Article CAS PubMed Google Scholar
Malech, H. L. et al. Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc. Natl Acad. Sci. USA 94, 1213312138 (1997).
Article CAS PubMed PubMed Central Google Scholar
Morgan, R. A., Gray, D., Lomova, A. & Kohn, D. B. Hematopoietic stem cell gene therapy: progress and lessons learned. Cell Stem Cell 21, 574590 (2017).
Article CAS PubMed PubMed Central Google Scholar
Sago, C. D. et al. Nanoparticles that deliver RNA to bone marrow identified by in vivo directed evolution. J. Am. Chem. Soc. 140, 1709517105 (2018).
Article CAS PubMed PubMed Central Google Scholar
Shi, D., Toyonaga, S. & Anderson, D. G. In vivo RNA delivery to hematopoietic stem and progenitor cells via targeted lipid nanoparticles. Nano Lett. 23, 29382944 (2023).
Article CAS PubMed PubMed Central Google Scholar
Sou, K., Goins, B., Oyajobi, B. O., Travi, B. L. & Phillips, W. T. Bone marrow-targeted liposomal carriers. Expert Opin. Drug Deliv. 8, 317328 (2011).
Article CAS PubMed PubMed Central Google Scholar
Sou, K., Klipper, R., Goins, B., Tsuchida, E. & Phillips, W. T. Circulation kinetics and organ distribution of Hb-vesicles developed as a red blood cell substitute. J. Pharmacol. Exp. Ther. 312, 702709 (2005).
Article CAS PubMed Google Scholar
Xue, L. et al. Rational design of bisphosphonate lipid-like materials for mRNA delivery to the bone microenvironment. J. Am. Chem. Soc. 144, 99269937 (2022).
Article CAS PubMed Google Scholar
Boulais, P. E. & Frenette, P. S. Making sense of hematopoietic stem cell niches. Blood 125, 26212629 (2015).
Article CAS PubMed PubMed Central Google Scholar
Ikonomi, N., Kuhlwein, S. D., Schwab, J. D. & Kestler, H. A. Awakening the HSC: dynamic modeling of HSC maintenance unravels regulation of the TP53 pathway and quiescence. Front. Physiol. 11, 848 (2020).
Article PubMed PubMed Central Google Scholar
Li, J. Quiescence regulators for hematopoietic stem cell. Exp. Hematol. 39, 511520 (2011).
Article PubMed Google Scholar
Man, Y., Yao, X., Yang, T. & Wang, Y. Hematopoietic stem cell niche during homeostasis, malignancy, and bone marrow transplantation. Front. Cell Dev. Biol. 9, 621214 (2021).
Article PubMed PubMed Central Google Scholar
Nakamura-Ishizu, A., Takizawa, H. & Suda, T. The analysis, roles and regulation of quiescence in hematopoietic stem cells. Development 141, 46564666 (2014).
Article CAS PubMed Google Scholar
Eppert, K. et al. Stem cell gene expression programs influence clinical outcome in human leukemia. Nat. Med. 17, 10861093 (2011).
Article CAS PubMed Google Scholar
Lapidot, T. et al. A cell initiating human acute myeloid leukaemia after transplantation into SCID mice. Nature 367, 645648 (1994).
Article CAS PubMed Google Scholar
Mandal, T., Beck, M., Kirsten, N., Linden, M. & Buske, C. Targeting murine leukemic stem cells by antibody functionalized mesoporous silica nanoparticles. Sci. Rep. 8, 989 (2018).
Article PubMed PubMed Central Google Scholar
Pei, S. & Jordan, C. T. How close are we to targeting the leukemia stem cell? Best Pract. Res. Clin. Haematol. 25, 415418 (2012).
Article CAS PubMed Google Scholar
Li, C. et al. Prophylactic in vivo hematopoietic stem cell gene therapy with an immune checkpoint inhibitor reverses tumor growth in syngeneic mouse tumor models. Cancer Res. 80, 549560 (2020).
Article CAS PubMed Google Scholar
Li, C. et al. In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal globin in beta-YAC mice. Blood Adv. 5, 11221135 (2021).
Article CAS PubMed PubMed Central Google Scholar
Li, C. et al. In vivo HSC gene therapy using a bi-modular HDAd5/35++ vector cures sickle cell disease in a mouse model. Mol. Ther. 29, 822837 (2021).
Article CAS PubMed Google Scholar
Li, C. et al. Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors. Mol. Ther. Methods Clin. Dev. 24, 127141 (2022).
Article PubMed Google Scholar
Psatha, N. et al. Enhanced HbF reactivation by multiplex mutagenesis of thalassemic CD34+ cells in vitro and in vivo. Blood 138, 15401553 (2021).
Article CAS PubMed PubMed Central Google Scholar
Muruve, D. A., Barnes, M. J., Stillman, I. E. & Libermann, T. A. Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo. Hum. Gene Ther. 10, 965976 (1999).
Article CAS PubMed Google Scholar
Sweeney, C. L. & De Ravin, S. S. The promise of in vivo HSC prime editing. Blood 141, 20392040 (2023).
Article CAS PubMed Google Scholar
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, 3744 (1997).
Article CAS PubMed Google Scholar
Lek, A. et al. Death after high-dose rAAV9 gene therapy in a patient with Duchennes muscular dystrophy. N. Engl. J. Med. 389, 12031210 (2023).
Article CAS PubMed Google Scholar
Hou, X., Zaks, T., Langer, R. & Dong, Y. Lipid nanoparticles for mRNA delivery. Nat. Rev. Mater. 6, 10781094 (2021).
Article CAS PubMed PubMed Central Google Scholar
Cheng, Q. et al. Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-Cas gene editing. Nat. Nanotechnol. 15, 313320 (2020).
Article CAS PubMed PubMed Central Google Scholar
Dilliard, S. A., Cheng, Q. & Siegwart, D. J. On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles. Proc. Natl Acad. Sci. USA 118, e2109256118 (2021).
Article CAS PubMed PubMed Central Google Scholar
Dilliard, S. A. & Siegwart, D. J. Passive, active and endogenous organ-targeted lipid and polymer nanoparticles for delivery of genetic drugs. Nat. Rev. Mater. 8, 282300 (2023).
Article CAS PubMed PubMed Central Google Scholar
Farbiak, L. et al. All-in-one dendrimer-based lipid nanoparticles enable precise HDR-mediated gene editing in vivo. Adv. Mater. 33, e2006619 (2021).
Article PubMed PubMed Central Google Scholar
Liu, S. et al. Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR-Cas gene editing. Nat. Mater. 20, 701710 (2021).
Article CAS PubMed PubMed Central Google Scholar
Liu, S. et al. Zwitterionic phospholipidation of cationic polymers facilitates systemic mRNA delivery to spleen and lymph nodes. J. Am. Chem. Soc. 143, 2132121330 (2021).
Article CAS PubMed PubMed Central Google Scholar
Wang, X. et al. Preparation of selective organ-targeting (SORT) lipid nanoparticles (LNPs) using multiple technical methods for tissue-specific mRNA delivery. Nat. Protoc. 18, 265291 (2023).
Article CAS PubMed Google Scholar
Wei, T., Cheng, Q., Min, Y. L., Olson, E. N. & Siegwart, D. J. Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing. Nat. Commun. 11, 3232 (2020).
Article CAS PubMed PubMed Central Google Scholar
Zhang, D. et al. Enhancing CRISPR/Cas gene editing through modulating cellular mechanical properties for cancer therapy. Nat. Nanotechnol. 17, 777787 (2022).
Article CAS PubMed PubMed Central Google Scholar
Wu, L. C. et al. Correction of sickle cell disease by homologous recombination in embryonic stem cells. Blood 108, 11831188 (2006).
Article CAS PubMed PubMed Central Google Scholar
Metais, J. Y. et al. Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv. 3, 33793392 (2019).
Article PubMed PubMed Central Google Scholar
Newby, G. A. et al. Base editing of haematopoietic stem cells rescues sickle cell disease in mice. Nature 595, 295302 (2021).
Article CAS PubMed PubMed Central Google Scholar
Stavropoulou, V., Peters, A. & Schwaller, J. Aggressive leukemia driven by MLL-AF9. Mol. Cell Oncol. 5, e1241854 (2018).
Article PubMed Google Scholar
Hou, X. et al. Vitamin lipid nanoparticles enable adoptive macrophage transfer for the treatment of multidrug-resistant bacterial sepsis. Nat. Nanotechnol. 15, 4146 (2020).
Article CAS PubMed PubMed Central Google Scholar
- Ancient viruses in the human genome linked to mental health conditions - New Scientist - May 23rd, 2024 [May 23rd, 2024]
- Study finds gene variants tied to breast cancer risk in Black women - STAT - May 23rd, 2024 [May 23rd, 2024]
- Active transcription and epigenetic reactions synergistically regulate meso-scale genomic organization - Nature.com - May 23rd, 2024 [May 23rd, 2024]
- The Human Pangenome: A Step Towards More Inclusive Genomic Medicine - Foley & Lardner LLP - May 23rd, 2024 [May 23rd, 2024]
- Genome-wide identification and analysis of epithelial-mesenchymal transition-related RNA-binding proteins and ... - Nature.com - May 23rd, 2024 [May 23rd, 2024]
- AI and CRISPR: Revolutionizing Genome Editing and Precision Medicine - MarkTechPost - May 23rd, 2024 [May 23rd, 2024]
- This AI Paper Introduces Evo: A Genomic Foundation Model that Enables Prediction and Generation Tasks from the Molecular to Genome-Scale -... - May 23rd, 2024 [May 23rd, 2024]
- Inocras and IMBdx Announce Strategic Partnership to Transform Cancer Patient Care in the U.S. - Morningstar - May 23rd, 2024 [May 23rd, 2024]
- Two Notes Genome review could this no-nonsense guitar plugin be all you need? - Guitar.com - May 23rd, 2024 [May 23rd, 2024]
- Research improves multiplex mutagenesis to increase experimental efficiency in plant genome editing - Phys.org - May 23rd, 2024 [May 23rd, 2024]
- Next Gen Diagnostics and Vanderbilt Report the Use of Whole Genome Sequencing to Detect Transmission of Infection ... - Newswire - May 23rd, 2024 [May 23rd, 2024]
- Genomic Enumeration of Antibiotic Resistance in Space (GEARS) Completes Mission Operations - Astrobiology - Astrobiology News - May 23rd, 2024 [May 23rd, 2024]
- Genome Editing Market size is set to grow by USD 7.23 billion from 2024-2028, Rapid technological advancements in ... - PR Newswire - May 23rd, 2024 [May 23rd, 2024]
- The complete plastome sequences of invasive weed Parthenium hysterophorus: genome organization, evolutionary ... - Nature.com - February 18th, 2024 [February 18th, 2024]
- Multi-omic profiling reveals associations between the gut microbiome, host genome and transcriptome in patients with ... - Journal of Translational... - February 18th, 2024 [February 18th, 2024]
- Polymerase Chain Reaction (PCR) - National Human Genome Research Institute - February 18th, 2024 [February 18th, 2024]
- Genomic Time Machine Reveals Secrets of Human DNA - SciTechDaily - February 18th, 2024 [February 18th, 2024]
- 1 Million Unannotated Exons Discovered in the Human Genome - Technology Networks - February 18th, 2024 [February 18th, 2024]
- Hope for the night parrot: bird's full genome has been sequenced - Cosmos - February 18th, 2024 [February 18th, 2024]
- RevIT AAV Enhancer: Rev-up AAV genome production in upstream manufacturing - BioProcess Insider - February 18th, 2024 [February 18th, 2024]
- Multi-omics resources for the Australian southern stuttering frog (Mixophyes australis) reveal assorted antimicrobial ... - Nature.com - February 18th, 2024 [February 18th, 2024]
- Large-scale gene expression alterations introduced by structural variation drive morphotype diversification in Brassica ... - Nature.com - February 18th, 2024 [February 18th, 2024]
- Near-gapless and haplotype-resolved apple genomes provide insights into the genetic basis of rootstock-induced ... - Nature.com - February 18th, 2024 [February 18th, 2024]
- Secrets of Night Parrot unlocked after first genome sequenced - CSIRO - February 18th, 2024 [February 18th, 2024]
- CRISPR gene editing tool gets a revolutionary high-tech upgrade - Earth.com - February 18th, 2024 [February 18th, 2024]
- Ancient retroviruses played a key role in the evolution of vertebrate brains - EurekAlert - February 18th, 2024 [February 18th, 2024]
- Natural selection and genetic diversity maintenance in a parasitic wasp during continuous biological control application - Nature.com - February 18th, 2024 [February 18th, 2024]
- Hopes elusive parrots genome will provide answers - news.com.au - February 18th, 2024 [February 18th, 2024]
- MicroRNA is the master regulator of the genome researchers are learning how to treat disease by harnessing the ... - The Conversation - November 30th, 2023 [November 30th, 2023]
- "Ground-Breaking" Release of World's Largest Whole Genome Resource - Inside Precision Medicine - November 30th, 2023 [November 30th, 2023]
- Pangenome analysis reveals genomic variations associated with domestication traits in broomcorn millet - Nature.com - November 30th, 2023 [November 30th, 2023]
- Global genetic diversity, introgression, and evolutionary adaptation of indicine cattle revealed by whole genome ... - Nature.com - November 30th, 2023 [November 30th, 2023]
- Genome characteristics of atypical porcine pestivirus from abortion cases in Shandong Province, China - Virology Journal - Virology Journal - November 30th, 2023 [November 30th, 2023]
- Correcting modification-mediated errors in nanopore sequencing by nucleotide demodification and reference-based ... - Nature.com - November 30th, 2023 [November 30th, 2023]
- CRISPR-Based "Genome Shredding" Technique Shows Promise in Treating Glioblastoma - Inside Precision Medicine - November 30th, 2023 [November 30th, 2023]
- Genome wide analysis revealed conserved domains involved in the effector discrimination of bacterial type VI secretion ... - Nature.com - November 30th, 2023 [November 30th, 2023]
- TRISH to investigate the effects of spaceflight on the human genome, central nervous system - Odessa American - November 30th, 2023 [November 30th, 2023]
- The venom preceded the stinger: Genomic studies shed light on the origins of bee venom - EurekAlert - November 30th, 2023 [November 30th, 2023]
- Integrating genomic and multiomic data for Angelica sinensis provides insights into the evolution and biosynthesis of ... - Nature.com - November 30th, 2023 [November 30th, 2023]
- Genetic diversity and ancestry of the Khmuic-speaking ethnic groups ... - Nature.com - September 21st, 2023 [September 21st, 2023]
- Researchers to Apply Genome Analysis to Childhood Cancers; Goal ... - The Japan News - September 21st, 2023 [September 21st, 2023]
- How Bats' Genomes May Help Them Avoid Cancer and Survive ... - Technology Networks - September 21st, 2023 [September 21st, 2023]
- Longitudinal genomic surveillance of carriage and transmission of ... - Nature.com - September 21st, 2023 [September 21st, 2023]
- Whole genomes from bacteria collected at diagnostic units around ... - Nature.com - September 21st, 2023 [September 21st, 2023]
- Genome-wide identification of lncRNA & mRNA for T2DM | PGPM - Dove Medical Press - September 21st, 2023 [September 21st, 2023]
- Tasmanian tiger RNA is first to be recovered from an extinct animal - Nature.com - September 21st, 2023 [September 21st, 2023]
- Loneliness and depression: bidirectional mendelian randomization ... - Nature.com - September 21st, 2023 [September 21st, 2023]
- Rome Therapeutics adds $72 million to Series B round to harness ... - OutSourcing-Pharma.com - September 21st, 2023 [September 21st, 2023]
- Mystery of 'living fossil' tree frozen in time for 66 million years finally ... - Livescience.com - September 21st, 2023 [September 21st, 2023]
- Why the human genome could be healthcares holy grail - Yahoo Finance - May 4th, 2023 [May 4th, 2023]
- Scientists Compare Genomes of 240 Mammals to Understand Human DNA - The New York Times - May 4th, 2023 [May 4th, 2023]
- Genomes From 240 Mammalian Species Help Explain 100 Years Of Evolution And Human Disease - ABP Live - May 4th, 2023 [May 4th, 2023]
- 'Deletions' from the human genome may be what made us human - Yale News - May 4th, 2023 [May 4th, 2023]
- GeneDx Adds Buccal Swab as Non-Invasive Whole Genome ... - GlobeNewswire - May 4th, 2023 [May 4th, 2023]
- Whole-genome sequencing used to track down genes behind familial glioma - Medical Xpress - May 4th, 2023 [May 4th, 2023]
- Wiggly proteins guard the genome: Dynamic network in the pores of ... - Science Daily - May 4th, 2023 [May 4th, 2023]
- Genome-Wide Splicing Quantitative Expression Locus Analysis ... - Cancer Discovery - May 4th, 2023 [May 4th, 2023]
- Digital Genome Market is expand at a CAGR of 8.6% to reach USD ... - Digital Journal - May 4th, 2023 [May 4th, 2023]
- High School Students Learn the Basics of Base Editing to Cure GFP ... - University of California San Diego - May 4th, 2023 [May 4th, 2023]
- Genomic researchers gain access to CSIRO's AI-powered data ... - Microsoft - May 4th, 2023 [May 4th, 2023]
- Archaic hominin traits through the splicing lens - Nature.com - May 4th, 2023 [May 4th, 2023]
- Critical bug in genome sequencing device scores '10' on CVSS ratings - SC Media - May 4th, 2023 [May 4th, 2023]
- Novel Genomic Approach Ensures Better Diagnosis of Hereditary ... - Technology Networks - May 4th, 2023 [May 4th, 2023]
- Intellia Therapeutics: Leading the Way in Revolutionary Genome ... - Best Stocks - May 4th, 2023 [May 4th, 2023]
- Visual tracking of viral infection dynamics reveals the synergistic ... - Nature.com - May 4th, 2023 [May 4th, 2023]
- Genome | Genome LLC | United States - March 31st, 2023 [March 31st, 2023]
- Belarus: EU and WHO deliver equipment for research of genomes of infectious disease agents - EIN News - February 24th, 2023 [February 24th, 2023]
- Gene vs. genome: Definition, function, and impact - January 30th, 2023 [January 30th, 2023]
- Big cog in the wheel: As Covid worries reappear, Insacogs genome sequencing ability must be aided by govts - Times of India - December 25th, 2022 [December 25th, 2022]
- CapitalGainsReport Sector Spotlight: Healthcare Penny Stocks On The Move (ARDX, WHSI, BNGO) - Marketscreener.com - November 25th, 2022 [November 25th, 2022]
- Genome Insight and Kun-hee Lee Child Cancer & Rare Disease Project Team of SNUH (Seoul National University Hospital) Made an Agreement About a... - November 23rd, 2022 [November 23rd, 2022]
- Genome-wide association study reveals distinct genetic associations related to leaf hair density in two lineages of wheat-wild relative Aegilops... - October 19th, 2022 [October 19th, 2022]
- The Global Genomics Market to Exhibit Growth at a CAGR of 16.90% During the Forecast Period (20222027) | DelveInsight - Yahoo Finance - October 19th, 2022 [October 19th, 2022]
- Illumina and GenoScreen Partner to Expand Access to Genomic Testing for Multidrug-Resistant Tuberculosis - PR Newswire - October 19th, 2022 [October 19th, 2022]
- Superresolution Method Poised to Better Gene Function Understanding - Photonics.com - October 19th, 2022 [October 19th, 2022]
- Genome-centric analysis of short and long read metagenomes reveals uncharacterized microbiome diversity in Southeast Asians - Nature.com - October 15th, 2022 [October 15th, 2022]
- How a New Battery Data Genome Project Will Use Vast Amounts of Information to Build Better EVs - InsideClimate News - October 15th, 2022 [October 15th, 2022]
- Scientists Reconstruct the Genome of the 180-Million-Year-Old Common Ancestor of All Mammals - SciTechDaily - October 15th, 2022 [October 15th, 2022]
- Combining OSMAC, metabolomic and genomic methods for the production and annotation of halogenated azaphilones and ilicicolins in termite symbiotic... - October 15th, 2022 [October 15th, 2022]
- Concerted expansion and contraction of immune receptor gene repertoires in plant genomes - Nature.com - October 15th, 2022 [October 15th, 2022]