Levesque, S. & Bauer, D. E. CRISPR-based therapeutic genome enhancing for inherited blood issues. Nat. Rev. Drug Discov. (2025).
Google ScholarÂ
Raguram, A., Banskota, S. & Liu, D. R. Therapeutic in vivo supply of gene enhancing brokers. Cell 185, 2806–2827 (2022).
Google ScholarÂ
Tsuchida, C. A., Wasko, Okay. M., Hamilton, J. R. & Doudna, J. A. Focused nonviral supply of genome editors in vivo. Proc. Natl Acad. Sci. USA 121, e2307796121 (2024).
Google ScholarÂ
Stigzelius, V., Cavallo, A. L., Chandode, R. Okay. & Nitsch, R. Peeling again the layers of immunogenicity in Cas9-based genomic medication. Mol. Ther. 33, 4714–4730 (2025).
Google ScholarÂ
Porteus, M. Genome enhancing: a brand new method to human therapeutics. Annu. Rev. Pharmacol. Toxicol. 56, 163–190 (2014).
Google ScholarÂ
Kumar, M., Kulkarni, P., Liu, S., Chemuturi, N. & Shah, D. Okay. Nanoparticle biodistribution coefficients: a quantitative method for understanding the tissue distribution of nanoparticles. Adv. Drug Deliv. Rev. 194, 114708 (2023).
Google ScholarÂ
Gillmore, J. D. et al. CRISPR–Cas9 in vivo gene enhancing for transthyretin amyloidosis. N. Engl. J. Med. 385, 493–502 (2021).
Google ScholarÂ
Musunuru, Okay. et al. Affected person-specific in vivo gene enhancing to deal with a uncommon genetic illness. N. Engl. J. Med. 392, 2235–2243 (2025).
Google ScholarÂ
Horie, T. & Ono, Okay. VERVE-101: a promising CRISPR-based gene enhancing remedy that reduces LDL-C and PCSK9 ranges in HeFH sufferers. Eur. Coronary heart J. Cardiovasc. Pharmacother. 10, 89–90 (2023).
Google ScholarÂ
Cohn, D. M. et al. CRISPR-based remedy for hereditary angioedema. N. Engl. J. Med. 392, 458–467 (2025).
Google ScholarÂ
Lee, R. et al. An investigational in vivo base enhancing medication concentrating on ANGPTL3, VERVE-201, achieves exact and sturdy liver enhancing in nonclinical research. Atherosclerosis 395, 118496 (2024).
Google ScholarÂ
Beam Therapeutics. A part 1/2 dose-exploration and dose-expansion examine to judge the protection and efficacy of BEAM-302 in grownup sufferers with α-1 antitrypsin deficiency (AATD)-associated lung illness and/or liver illness. ClinicalTrials.gov (2024).
Morrow, P. Okay. et al. Summary 17013: CTX320: an investigational in vivo CRISPR-based remedy effectively and durably reduces lipoprotein (a) ranges in non-human primates after a single dose. Circulation 148, A17013 (2023).
HuidaGene Therapeutics. An investigator-initiated scientific examine evaluating the CRISPR–hfCas12Max gene enhancing remedy within the therapy of Duchenne muscular dystrophy (DMD). ClinicalTrials.gov (2024).
Beam Therapeutics. A part 1/2, dose-exploration examine to judge the protection and efficacy of BEAM-301 in sufferers with glycogen storage illness kind Ia (GSDIa) homozygous or compound heterozygous for the G6PC1 c.247C>T (p.R83C) variant. ClinicalTrials.gov (2024).
Arbor Biotechnologies. A part 1/2 dose escalation examine to judge the protection, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of ABO-101 in individuals with main hyperoxaluria kind 1 (PH1). ClinicalTrials.gov (2025).
Tune Therapeutics. Part 1b multicenter, open-label examine to evaluate the protection, tolerability, pharmacokinetics, and pharmacodynamics of Tune-401 in individuals with power hepatitis B an infection. ClinicalTrials.gov (2024).
Burdo, T. H. et al. Preclinical security and biodistribution of CRISPR concentrating on SIV in non-human primates. Gene Ther. 31, 224–233 (2023).
Google ScholarÂ
Excision BioTherapeutics. A part 1/2a, sequential cohort, single ascending dose examine of the protection, tolerability, biodistribution, and pharmacodynamics of EBT 101 in aviremic HIV-1 contaminated adults on secure antiretroviral remedy. ClinicalTrials.gov (2021).
Epicrispr Biotechnologies. A part 1/2 open-label dose-escalation examine to judge the protection, tolerability, and organic exercise of EPI-321, an AAVrh74-delivered epigenetic enhancing remedy in grownup FSHD sufferers. ClinicalTrials.gov (2025).
Streilein, J. W. Ocular immune privilege: therapeutic alternatives from an experiment of nature. Nat. Rev. Immunol. 3, 879–889 (2003).
Google ScholarÂ
Nakao, S., Hafezi-Moghadam, A. & Ishibashi, T. Lymphatics and lymphangiogenesis within the eye. J. Ophthalmol. 2012, 783163 (2012).
Google ScholarÂ
Toral, M. A. et al. Investigation of Cas9 antibodies within the human eye. Nat. Commun. 13, 1053 (2022).
Google ScholarÂ
Pierce, E. A. et al. Gene enhancing for CEP290-associated retinal degeneration. N. Engl. J. Med. 390, 1972–1984 (2024).
Google ScholarÂ
Zhao, Q., Wei, L. & Chen, Y. From bench to bedside: creating CRISPR/Cas-based remedy for ocular ailments. Pharmacol. Res. 213, 107638 (2025).
Google ScholarÂ
Muller, A. et al. Excessive-efficiency base enhancing within the retina in primates and human tissues. Nat. Med. 31, 490–501 (2025).
Google ScholarÂ
Luk, A. et al. World’s first CRISPR/RNA-targeting remedy (HG202) for sufferers with neovascular age-related macular degeneration. Make investments. Ophthalmol. Vis. Sci. 65, 4357 (2024).
Wei, A. et al. In vivo CRISPR gene enhancing in sufferers with herpetic stromal keratitis. Mol. Ther. 31, 3163–3175 (2023).
Google ScholarÂ
Jain, A. et al. CRISPR–Cas9–primarily based therapy of myocilin-associated glaucoma. Proc. Natl Acad. Sci. USA 114, 11199–11204 (2017).
Google ScholarÂ
Gencay, Y. E. et al. Engineered phage with antibacterial CRISPR–Cas selectively scale back E. coli burden in mice. Nat. Biotechnol. 42, 265–274 (2024).
Google ScholarÂ
SNIPR Biome. A part 1, randomized, double-blind, first-in-human, dose escalation examine investigating the protection, restoration, and pharmacodynamics of a number of oral administrations of SNIPR001 in wholesome topics. ClinicalTrials.gov (2022).
SNIPR Biome. SNIPR Biome Stories Optimistic Scientific Interim Outcomes for Groundbreaking, First-in-Human, CRISPR-Primarily based Microbial Gene Remedy (2023).
Xue, Y. et al. RNA base enhancing remedy cures listening to loss induced by OTOF gene mutation. Mol. Ther. 31, 3520–3530 (2023).
Google ScholarÂ
HuidaGene Therapeutics. An open-label, multiple-cohort, dose-finding, investigator-initiated trial to judge the protection, tolerability, and efficacy of HG205 RNA base-editing remedy in topics with OTOF-p.Q829X mutation-associated listening to loss. ClinicalTrials.gov (2023).
Yang, D. et al. An RNA enhancing technique rescues gene duplication in a mouse mannequin of MECP2 duplication syndrome and nonhuman primates. Nat. Neurosci. 28, 72–83 (2025).
Google ScholarÂ
HuidaGene Therapeutics. An open-label, multiple-dose scientific examine to evaluating the protection, tolerability and preliminary efficacy of a single intracerebroventricular injection of HG204 for the therapy of MECP2 duplication syndrome. ClinicalTrials.gov (2024).
Lenneman, B. R., Fernbach, J., Loessner, M. J., Lu, T. Okay. & Kilcher, S. Enhancing phage remedy by artificial biology and genome engineering. Curr. Opin. Biotechnol. 68, 151–159 (2021).
Google ScholarÂ
Kim, P. et al. Security, pharmacokinetics, and pharmacodynamics of LBP-EC01, a CRISPR–Cas3-enhanced bacteriophage cocktail, in uncomplicated urinary tract infections because of Escherichia coli (ELIMINATE): the randomised, open-label, first a part of a two-part part 2 trial. Lancet Infect. Dis. 24, 1319–1332 (2024).
Google ScholarÂ
Amoasii, L. et al. Single-cut genome enhancing restores dystrophin expression in a brand new mouse mannequin of muscular dystrophy. Sci. Transl. Med. 9, eaan8081 (2017).
Google ScholarÂ
Ho, T.-C. et al. Scaffold-mediated CRISPR–Cas9 supply system for acute myeloid leukemia remedy. Sci. Adv. 7, eabg3217 (2021).
Google ScholarÂ
Liang, S.-Q. et al. AAV5 supply of CRISPR–Cas9 helps efficient genome enhancing in mouse lung airway. Mol. Ther. 30, 238–243 (2022).
Google ScholarÂ
Rosenblum, D. et al. CRISPR–Cas9 genome enhancing utilizing focused lipid nanoparticles for most cancers remedy. Sci. Adv. 6, eabc9450 (2020).
Google ScholarÂ
Stahl, E. C. et al. Genome enhancing within the mouse mind with minimally immunogenic Cas9 RNPs. Mol. Ther. 31, 2422–2438 (2023).
Google ScholarÂ
Kasiewicz, L. N. et al. GalNAc-lipid nanoparticles allow non-LDLR dependent hepatic supply of a CRISPR base enhancing remedy. Nat. Commun. 14, 2776 (2023).
Google ScholarÂ
Lee, R. et al. An investigational in vivo base enhancing medication concentrating on ANGPTL3, VERVE-201, achieves potent and LDLR-independent liver enhancing in mouse fashions. Eur. Coronary heart J. 44, ehad655.2521 (2023).
Google ScholarÂ
Verve Therapeutics. A part 1b single ascending dose examine to judge the protection of VERVE-201 in sufferers with refractory hyperlipidemia. ClinicalTrials.gov (2024).
Pupo, A. et al. AAV vectors: the Rubik’s dice of human gene remedy. Mol. Ther. 30, 3515–3541 (2022).
Google ScholarÂ
Gao, G.-P. et al. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene remedy. Proc. Natl Acad. Sci. USA 99, 11854–11859 (2002).
Google ScholarÂ
Strebinger, D. et al. Cell type-specific supply by modular envelope design. Nat. Commun. 14, 5141 (2023).
Google ScholarÂ
Hamilton, J. R. et al. In vivo human T cell engineering with enveloped supply automobiles. Nat. Biotechnol. 42, 1684–1692 (2024).
Google ScholarÂ
Hamilton, J. R. et al. Focused supply of CRISPR–Cas9 and transgenes allows complicated immune cell engineering. Cell Rep. 35, 109207 (2021).
Google ScholarÂ
Ngo, W. et al. Mechanism-guided engineering of a minimal organic particle for genome enhancing. Proc. Natl Acad. Sci. USA 122, e2413519121 (2025).
Google ScholarÂ
Karp, H. et al. Packaged supply of CRISPR–Cas9 ribonucleoproteins accelerates genome enhancing. Nucleic Acids Res. 53, gkaf105 (2025).
Google ScholarÂ
Breda, L. et al. In vivo hematopoietic stem cell modification by mRNA supply. Science 381, 436–443 (2023).
Google ScholarÂ
Palanki, R. et al. In utero supply of focused ionizable lipid nanoparticles facilitates in vivo gene enhancing of hematopoietic stem cells. Proc. Natl Acad. Sci. USA 121, e2400783121 (2024).
Google ScholarÂ
Geczy, R. et al. Lipid nanoparticle-mediated gene enhancing of human main T cells and off-target evaluation of the CRISPR–Cas9 indels. Blood 142, 6833 (2023).
Google ScholarÂ
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).
Google ScholarÂ
Cheng, Q. et al. Selective organ concentrating on (SORT) nanoparticles for tissue particular mRNA supply and CRISPR/Cas gene enhancing. Nat. Nanotechnol. 15, 313–320 (2020).
Google ScholarÂ
Chen, Okay. et al. Lung and liver enhancing by lipid nanoparticle supply of a secure CRISPR–Cas9 ribonucleoprotein. Nat. Biotechnol. 43, 1445–1457 (2025).
Google ScholarÂ
Kimura, S. & Harashima, H. On the mechanism of tissue-selective gene supply by lipid nanoparticles. J. Management. Launch 362, 797–811 (2023).
Google ScholarÂ
Tabebordbar, M. et al. Directed evolution of a household of AAV capsid variants enabling potent muscle-directed gene supply throughout species. Cell 184, 4919–4938 (2021).
Google ScholarÂ
Huang, Q. et al. An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene supply. Science 384, 1220–1227 (2024).
Google ScholarÂ
Neumann, E. N. et al. Brainwide silencing of prion protein by AAV-mediated supply of an engineered compact epigenetic editor. Science 384, ado7082 (2024).
Google ScholarÂ
Kumar, S. R. et al. Multiplexed Cre-dependent choice yields systemic AAVs for concentrating on distinct mind cell sorts. Nat. Strategies 17, 541–550 (2020).
Google ScholarÂ
Kim, H. et al. Lipid nanoparticle-mediated mRNA supply to CD34+ cells in rhesus monkeys. Nat. Biotechnol. 43, 1813–1820 (2024).
Google ScholarÂ
Dahlman, J. E. et al. Barcoded nanoparticles for top throughput in vivo discovery of focused therapeutics. Proc. Natl Acad. Sci. USA 114, 2060–2065 (2017).
Google ScholarÂ
Ngo, W. et al. Why nanoparticles choose liver macrophage cell uptake in vivo. Adv. Drug Deliv. Rev. 185, 114238 (2022).
Google ScholarÂ
Glaumann, H., Fredzell, J., Jubner, A. & Ericsson, J. L. E. Uptake and degradation of glycogen by Kupffer cells. Exp. Mol. Pathol. 31, 70–80 (1979).
Google ScholarÂ
Web optimization, J. W. et al. Multimodal imaging of capsid and cargo reveals differential mind concentrating on and liver detargeting of systemically-administered AAVs. Biomaterials 288, 121701 (2022).
Google ScholarÂ
l’Hortet, A. C. et al. In MDA Scientific & Scientific Convention 206 (Muscular Dystrophy Affiliation, 2023).
Amoasii, L. et al. Gene enhancing restores dystrophin expression in a canine mannequin of Duchenne muscular dystrophy. Science 362, 86–91 (2018).
Google ScholarÂ
Vaessen, S. F. C. et al. AAV gene remedy as a way to extend apolipoprotein (Apo) A-I and high-density lipoprotein-cholesterol ranges: correction of murine ApoA-I deficiency. J. Gene Med. 11, 697–707 (2009).
Google ScholarÂ
Prasad, Okay.-M. R., Xu, Y., Yang, Z., Acton, S. T. & French, B. A. Strong cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene supply follows a Poisson distribution. Gene Ther. 18, 43–52 (2011).
Google ScholarÂ
Radhiyanti, P. T., Konno, A., Matsuzaki, Y. & Hirai, H. Comparative examine of neuron-specific promoters in mouse mind transduced by intravenously administered AAV-PHP.eB. Neurosci. Lett. 756, 135956 (2021).
Google ScholarÂ
Yang, L. et al. MicroRNA-122-mediated liver detargeting enhances the tissue specificity of cardiac genome enhancing. Circulation 149, 1778–1781 (2024).
Google ScholarÂ
Hoffmann, M. D. et al. Cell-specific CRISPR–Cas9 activation by microRNA-dependent expression of anti-CRISPR proteins. Nucleic Acids Res. 47, e75 (2019).
Google ScholarÂ
Hirosawa, M., Fujita, Y. & Saito, H. Cell-type-specific CRISPR activation with microRNA-responsive AcrllA4 change. ACS Synth. Biol. 8, 1575–1582 (2019).
Google ScholarÂ
Lee, J. et al. Tissue-restricted genome enhancing in vivo specified by microRNA-repressible anti-CRISPR proteins. RNA 25, 1421–1431 (2019).
Google ScholarÂ
Wang, X.-W. et al. A microRNA-inducible CRISPR–Cas9 platform serves as a microRNA sensor and cell-type-specific genome regulation software. Nat. Cell Biol. 21, 522–530 (2019).
Google ScholarÂ
Garcia-Guerra, A. et al. Tissue-specific modulation of CRISPR exercise by miRNA-sensing information RNAs. Nucleic Acids Res. 53, gkaf016 (2025).
Google ScholarÂ
Galizi, R. & Jaramillo, A. Engineering CRISPR information RNA riboswitches for in vivo purposes. Curr. Opin. Biotechnol. 55, 103–113 (2019).
Google ScholarÂ
Kaseniit, Okay. E. et al. Modular, programmable RNA sensing utilizing ADAR enhancing in dwelling cells. Nat. Biotechnol. 41, 482–487 (2023).
Google ScholarÂ
Jiang, Okay. et al. Programmable eukaryotic protein synthesis with RNA sensors by harnessing ADAR. Nat. Biotechnol. 41, 698–707 (2023).
Google ScholarÂ
Qian, Y. et al. Programmable RNA sensing for cell monitoring and manipulation. Nature 610, 713–721 (2022).
Google ScholarÂ
Powell, S. Okay., Rivera-Soto, R. & Grey, S. J. Viral expression cassette parts to boost transgene goal specificity and expression in gene remedy. Discov. Med. 19, 49–57 (2015).
Google ScholarÂ
Mancuso, P. et al. CRISPR primarily based enhancing of SIV proviral DNA in ART handled non-human primates. Nat. Commun. 11, 6065 (2020).
Google ScholarÂ
Cohrt, Okay. O. Excision’s EBT-101 demonstrates security in scientific trial however doesn’t treatment HIV. CRISPR Medication Information (2024).
Tan, I.-L. et al. Focusing on the non-coding genome and temozolomide signature allows CRISPR-mediated glioma oncolysis. Cell Rep. 42, 113339 (2023).
Google ScholarÂ
An, Y. et al. Design of hypoxia responsive CRISPR–Cas9 for goal gene regulation. Sci. Rep. 13, 16763 (2023).
Google ScholarÂ
Chen, X., Chen, Y., Xin, H., Wan, T. & Ping, Y. Close to-infrared optogenetic engineering of photothermal nanoCRISPR for programmable genome enhancing. Proc. Natl Acad. Sci. USA 117, 2395–2405 (2020).
Google ScholarÂ
Yin, H. et al. Ultrasound-controlled CRISPR/Cas9 system augments sonodynamic remedy of hepatocellular carcinoma. ACS Cent. Sci. 7, 2049–2062 (2021).
Google ScholarÂ
Liu, Y. et al. Very quick CRISPR on demand. Science 368, 1265–1269 (2020).
Google ScholarÂ
Pacesa, M. et al. Structural foundation for Cas9 off-target exercise. Cell 185, 4067–4081 (2022).
Google ScholarÂ
Greig, J. A. et al. Built-in vector genomes could contribute to long-term expression in primate liver after AAV administration. Nat. Biotechnol. 42, 1232–1242 (2024).
Google ScholarÂ
iECURE. A part 1/2/3 first-in-human, open-label, dose-escalation examine to judge the protection and efficacy of a single intravenous (IV) administration of ECUR-506 in males lower than 9 months of age with genetically confirmed neonatal onset ornithine transcarbamylase (OTC) deficiency. ClinicalTrials.gov (2023).
Regeneron Prescribed drugs. A two-part open-label examine of REGV131-LNP1265, a CRISPR/Cas9 primarily based coagulation issue IX gene insertion remedy in individuals with hemophilia B. ClinicalTrials.gov (2024).
Jeune, V. L., Joergensen, J. A., Hajjar, R. J. & Weber, T. Pre-existing anti-adeno-associated virus antibodies as a problem in AAV gene remedy. Hum. Gene Ther. Strategies 24, 59–67 (2013).
Google ScholarÂ
Duan, D. Deadly immunotoxicity in high-dose systemic AAV remedy. Mol. Ther. 31, 3123–3126 (2023).
Google ScholarÂ
Lee, Y., Jeong, M., Park, J., Jung, H. & Lee, H. Immunogenicity of lipid nanoparticles and its affect on the efficacy of mRNA vaccines and therapeutics. Exp. Mol. Med. 55, 2085–2096 (2023).
Google ScholarÂ
Vargas, J. E. et al. Retroviral vectors and transposons for secure gene remedy: advances, present challenges and views. J. Transl. Med. 14, 288 (2016).
Google ScholarÂ
Wignakumar, T. & Fairchild, P. J. Evasion of pre-existing immunity to Cas9: a prerequisite for profitable genome enhancing in vivo? Curr. Transplant. Rep. 6, 127–133 (2019).
Google ScholarÂ
Kishimoto, T. Okay. & Samulski, R. J. Addressing excessive dose AAV toxicity — ‘one and finished’ or ‘slower and decrease’? Professional Opin. Biol. Ther. 22, 1067–1071 (2022).
Google ScholarÂ
Cullis, P. R. & Hope, M. J. Lipid nanoparticle techniques for enabling gene therapies. Mol. Ther. 25, 1467–1475 (2017).
Google ScholarÂ
Cullis, P. R. & Felgner, P. L. The 60-year evolution of lipid nanoparticles for nucleic acid supply. Nat. Rev. Drug Discov. 23, 709–722 (2024).
Google ScholarÂ
Carbonaro-Sarracino, D. A. et al. Dosing and re-administration of intravenous lentiviral vector for liver-directed gene switch in younger rhesus monkeys and ADA-deficient mice. Mol. Ther. Strategies Clin. Dev. 24, S302–S303 (2016).
Chen, Okay. et al. Engineering self-deliverable ribonucleoproteins for genome enhancing within the mind. Nat. Commun. 15, 1727 (2024).
Google ScholarÂ
Staahl, B. T. et al. Environment friendly genome enhancing within the mouse mind by native supply of engineered Cas9 ribonucleoprotein complexes. Nat. Biotechnol. 35, 431–434 (2017).
Google ScholarÂ
Chew, W. L. Immunity to CRISPR Cas9 and Cas12a therapeutics. Wiley Interdiscip. Rev. Syst. Biol. Med. (2018).
Andari, J. E. & Grimm, D. Manufacturing, processing, and characterization of artificial AAV gene remedy vectors. Biotechnol. J. 16, e2000025 (2021).
Google ScholarÂ
Jiang, Z. & Dalby, P. A. Challenges in scaling up AAV-based gene remedy manufacturing. Traits Biotechnol. 41, 1268–1281 (2023).
Google ScholarÂ
De, A. & Ko, Y. T. Why mRNA-ionizable LNPs formulations are so short-lived: causes and way-out. Professional Opin. Drug Deliv. 20, 175–187 (2023).
Google ScholarÂ
Kim, B. et al. Optimization of storage situations for lipid nanoparticle-formulated self-replicating RNA vaccines. J. Management. Launch 353, 241–253 (2023).
Google ScholarÂ
Mangeot, P. E. et al. Genome enhancing in main cells and in vivo utilizing viral-derived Nanoblades loaded with Cas9–sgRNA ribonucleoproteins. Nat. Commun. 10, 45 (2019).
Google ScholarÂ
Merten, O.-W., Hebben, M. & Bovolenta, C. Manufacturing of lentiviral vectors. Mol. Ther. Strategies Clin. Dev. 3, 16017 (2016).
Google ScholarÂ
Binder, G. Okay. & Chen, C.-C. The very secure lentiviral vector. Mol. Ther. Strategies Clin. Dev. 32, 101223 (2024).
Google ScholarÂ
Berry, G. E. & Asokan, A. Mobile transduction mechanisms of adeno-associated viral vectors. Curr. Opin. Virol. 21, 54–60 (2016).
Google ScholarÂ
Patel, M. N. et al. Safer non-viral DNA supply utilizing lipid nanoparticles loaded with endogenous anti-inflammatory lipids. Nat. Biotechnol. (2025).
Google ScholarÂ
Banskota, S. et al. Engineered virus-like particles for environment friendly in vivo supply of therapeutic proteins. Cell 185, 250–265 (2022).
Google ScholarÂ
An, M. et al. Engineered virus-like particles for transient supply of prime editor ribonucleoprotein complexes in vivo. Nat. Biotechnol. 42, 1526–1537 (2024).
Google ScholarÂ
Lyu, P., Javidi-Parsijani, P., Atala, A. & Lu, B. Delivering Cas9/sgRNA ribonucleoprotein (RNP) by lentiviral capsid-based bionanoparticles for environment friendly ‘hit-and-run’ genome enhancing. Nucleic Acids Res. 47, e99 (2019).
Google ScholarÂ
Indikova, I. & Indik, S. Extremely environment friendly ‘hit-and-run’ genome enhancing with unconcentrated lentivectors carrying Vpr.Prot.Cas9 protein produced from RRE-containing transcripts. Nucleic Acids Res. 48, 8178–8187 (2020).
Google ScholarÂ
Gao, G., Vandenberghe, L. H. & Wilson, J. M. New recombinant serotypes of AAV vectors. Curr. Gene Ther. 5, 285–297 (2005).
Google ScholarÂ
Pham, Q. et al. A facile chemical technique to synthesize exact AAV-protein conjugates for focused gene supply. Mol. Ther. Oncol. 33, 201040 (2025).
Google ScholarÂ
Domenger, C. & Grimm, D. Subsequent-generation AAV vectors—don’t choose a virus (solely) by its cowl. Hum. Mol. Genet. 28, R3–R14 (2019).
Google ScholarÂ
Billingsley, M. M. et al. In vivo mRNA CAR T cell engineering by way of focused ionizable lipid nanoparticles with extrahepatic tropism. Small 20, e2304378 (2024).
Google ScholarÂ
Rurik, J. G. et al. CAR T cells produced in vivo to deal with cardiac damage. Science 375, 91–96 (2022).
Google ScholarÂ
Veiga, N. et al. Cell particular supply of modified mRNA expressing therapeutic proteins to leukocytes. Nat. Commun. 9, 4493 (2018).
Google ScholarÂ
Dobson, C. S. et al. Antigen identification and high-throughput interplay mapping by reprogramming viral entry. Nat. Strategies 19, 449–460 (2022).
Google ScholarÂ
Girard-Gagnepain, A. et al. Baboon envelope pseudotyped LVs outperform VSV-G-LVs for gene switch into early-cytokine-stimulated and resting HSCs. Blood 124, 1221–1231 (2014).
Google ScholarÂ
Seydel, C. Highlight Therapeutics: making CRISPR ship in vivo. Nat. Biotechnol. (2021).
Google ScholarÂ
Leave a Reply