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For: Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, Gross F, Yvon E, Nusbaum P, Selz F, Hue C, Certain S, Casanova JL, Bousso P, Deist FL, Fischer A. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000;288:669-72. [PMID: 10784449 DOI: 10.1126/science.288.5466.669] [Citation(s) in RCA: 1690] [Impact Index Per Article: 67.6] [Reference Citation Analysis] [What about the content of this article? (0)] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/11/2022]
Number Cited by Other Article(s)
1
Badal AK, Nayek A, Dhar R, Karmakar S. MicroRNA nanoformulation: a promising approach to anti-tumour activity. Invest New Drugs 2025:10.1007/s10637-025-01534-7. [PMID: 40366533 DOI: 10.1007/s10637-025-01534-7] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/30/2025] [Accepted: 04/17/2025] [Indexed: 05/15/2025]
2
Williams DA, Kohn DB, Thrasher AJ. Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy. Mol Ther 2025;33:2141-2153. [PMID: 40176348 DOI: 10.1016/j.ymthe.2025.03.058] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/20/2025] [Revised: 03/28/2025] [Accepted: 03/28/2025] [Indexed: 04/04/2025]  Open
3
Odio-Herrera M, Orozco-Loaiza G, Wu L. Gene Therapy in Diabetic Retinopathy and Diabetic Macular Edema: An Update. J Clin Med 2025;14:3205. [PMID: 40364236 PMCID: PMC12072420 DOI: 10.3390/jcm14093205] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/02/2025] [Revised: 04/22/2025] [Accepted: 04/28/2025] [Indexed: 05/15/2025]  Open
4
Hu M, Xu Q, Zhang F, Buckland KF, Gao Y, Du W, Ding Y, Zhou L, Sun X, Ma L, Zhang Z, Tang X, Zhao X, Thrasher AJ, An Y. Preclinical ex vivo IL2RG gene therapy using autologous hematopoietic stem cells as an effective and safe treatment for X-linked severe combined immunodeficiency disease. Genes Dis 2025;12:101445. [PMID: 40092492 PMCID: PMC11907444 DOI: 10.1016/j.gendis.2024.101445] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/08/2023] [Revised: 05/20/2024] [Accepted: 08/25/2024] [Indexed: 03/19/2025]  Open
5
Quesada E, Rojas S, Campos X, Wu L. Gene therapy in neovascular age related macular degeneration: an update. Graefes Arch Clin Exp Ophthalmol 2025:10.1007/s00417-025-06837-2. [PMID: 40293479 DOI: 10.1007/s00417-025-06837-2] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/30/2025] [Revised: 04/11/2025] [Accepted: 04/21/2025] [Indexed: 04/30/2025]  Open
6
Ottaviano G, Qasim W. Current landscape of vector safety and genotoxicity after hematopoietic stem or immune cell gene therapy. Leukemia 2025:10.1038/s41375-025-02585-8. [PMID: 40200078 DOI: 10.1038/s41375-025-02585-8] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/18/2024] [Revised: 03/10/2025] [Accepted: 03/21/2025] [Indexed: 04/10/2025]
7
Guthrie OW. Gene Therapy: An Historical Overview for Familial Hearing Loss. Int J Mol Sci 2025;26:1469. [PMID: 40003934 PMCID: PMC11855000 DOI: 10.3390/ijms26041469] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/19/2024] [Revised: 01/27/2025] [Accepted: 01/29/2025] [Indexed: 02/27/2025]  Open
8
Oliveira BC, Bari S, Melenhorst JJ. Leveraging Vector-Based Gene Disruptions to Enhance CAR T-Cell Effectiveness. Cancers (Basel) 2025;17:383. [PMID: 39941752 PMCID: PMC11815729 DOI: 10.3390/cancers17030383] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/17/2024] [Revised: 01/21/2025] [Accepted: 01/21/2025] [Indexed: 02/16/2025]  Open
9
Inamdar A, Gurupadayya B, Halagali P, Nandakumar S, Pathak R, Singh H, Sharma H. Cutting-edge Strategies for Overcoming Therapeutic Barriers in Alzheimer's Disease. Curr Pharm Des 2025;31:598-618. [PMID: 39492772 DOI: 10.2174/0113816128344571241018154506] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/04/2024] [Revised: 09/13/2024] [Accepted: 09/16/2024] [Indexed: 11/05/2024]
10
Gilioli G, Lankester AC, de Kivit S, Staal FJT, Ott de Bruin LM. Gene therapy strategies for RAG1 deficiency: Challenges and breakthroughs. Immunol Lett 2024;270:106931. [PMID: 39303994 DOI: 10.1016/j.imlet.2024.106931] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/23/2024] [Revised: 09/14/2024] [Accepted: 09/17/2024] [Indexed: 09/22/2024]
11
Torreggiani S, Castellan FS, Aksentijevich I, Beck DB. Somatic mutations in autoinflammatory and autoimmune disease. Nat Rev Rheumatol 2024;20:683-698. [PMID: 39394526 DOI: 10.1038/s41584-024-01168-8] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 09/05/2024] [Indexed: 10/13/2024]
12
Mrksich K, Padilla MS, Mitchell MJ. Breaking the final barrier: Evolution of cationic and ionizable lipid structure in lipid nanoparticles to escape the endosome. Adv Drug Deliv Rev 2024;214:115446. [PMID: 39293650 PMCID: PMC11900896 DOI: 10.1016/j.addr.2024.115446] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/05/2024] [Revised: 08/18/2024] [Accepted: 09/07/2024] [Indexed: 09/20/2024]
13
Kong C, Yin G, Wang X, Sun Y. In Utero Gene Therapy and its Application in Genetic Hearing Loss. Adv Biol (Weinh) 2024;8:e2400193. [PMID: 39007241 DOI: 10.1002/adbi.202400193] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/08/2024] [Revised: 07/03/2024] [Indexed: 07/16/2024]
14
Wang F, Huang Y, Li J, Zhou W, Wang W. Targeted gene delivery systems for T-cell engineering. Cell Oncol (Dordr) 2024;47:1537-1560. [PMID: 38753155 DOI: 10.1007/s13402-024-00954-6] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 04/28/2024] [Indexed: 06/27/2024]  Open
15
Tajer P, Karakaslar EO, Canté-Barrett K, Naber BAE, Vloemans SA, van Eggermond MCJA, van der Hoorn ML, van den Akker E, Pike-Overzet K, Staal FJT. Utilizing epigenetic regulators to improve HSC-based lentiviral gene therapy. Blood Adv 2024;8:4936-4947. [PMID: 38916861 PMCID: PMC11421325 DOI: 10.1182/bloodadvances.2024013047] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/29/2024] [Revised: 05/29/2024] [Accepted: 06/10/2024] [Indexed: 06/26/2024]  Open
16
Liu J, Li Y, Zhou K, Zhang S, Wang Y, Wang X, Lan X, Chen Q, Zhao Y. In Situ Polymerization for Manufacture of Multifunctional Delivery Systems for Transcellular Delivery of Nucleic Acids. Bioconjug Chem 2024;35:1417-1428. [PMID: 39225485 DOI: 10.1021/acs.bioconjchem.4c00319] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 09/04/2024]
17
Araujo AE, Bentler M, Perez Garmendia X, Kaleem A, Fabian C, Morgan M, Hacker UT, Büning H. Adeno-Associated Virus Vectors-a Target of Cellular and Humoral Immunity-are Expanding Their Reach Toward Hematopoietic Stem Cell Modification and Immunotherapies. Hum Gene Ther 2024;35:586-603. [PMID: 39193633 DOI: 10.1089/hum.2024.114] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 08/29/2024]  Open
18
Ha TC, Morgan MA, Thrasher AJ, Schambach A. Alpharetroviral Vector-Mediated Gene Therapy for IL7RA-Deficient Severe Combined Immunodeficiency. Hum Gene Ther 2024;35:669-679. [PMID: 39150017 DOI: 10.1089/hum.2024.103] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 08/17/2024]  Open
19
Arora Y, Priya, Kumar M, Kumar D. Current approaches in CRISPR-Cas system for metabolic disorder. PROGRESS IN MOLECULAR BIOLOGY AND TRANSLATIONAL SCIENCE 2024;210:1-19. [PMID: 39824577 DOI: 10.1016/bs.pmbts.2024.07.016] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 01/03/2025]
20
Taghdiri M, Mussolino C. Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets. Int J Mol Sci 2024;25:7333. [PMID: 39000440 PMCID: PMC11242246 DOI: 10.3390/ijms25137333] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/29/2024] [Revised: 06/10/2024] [Accepted: 06/24/2024] [Indexed: 07/16/2024]  Open
21
Cornel MC, van der Meij KRM, van El CG, Rigter T, Henneman L. Genetic Screening-Emerging Issues. Genes (Basel) 2024;15:581. [PMID: 38790210 PMCID: PMC11121342 DOI: 10.3390/genes15050581] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/29/2024] [Revised: 04/25/2024] [Accepted: 04/30/2024] [Indexed: 05/26/2024]  Open
22
Dhayalan M, Wang W, Riyaz SUM, Dinesh RA, Shanmugam J, Irudayaraj SS, Stalin A, Giri J, Mallik S, Hu R. Advances in functional lipid nanoparticles: from drug delivery platforms to clinical applications. 3 Biotech 2024;14:57. [PMID: 38298556 PMCID: PMC10825110 DOI: 10.1007/s13205-023-03901-8] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/28/2023] [Accepted: 12/18/2023] [Indexed: 02/02/2024]  Open
23
Singh H. Role of gene therapy in treatment of cancer with craniofacial regeneration-current molecular strategies, future perspectives, and challenges: a narrative review. JOURNAL OF YEUNGNAM MEDICAL SCIENCE 2024;41:13-21. [PMID: 37218144 PMCID: PMC10834268 DOI: 10.12701/jyms.2023.00073] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Subscribe] [Scholar Register] [Received: 01/26/2023] [Accepted: 03/30/2023] [Indexed: 05/24/2023]
24
Waddington SN, Peranteau WH, Rahim AA, Boyle AK, Kurian MA, Gissen P, Chan JKY, David AL. Fetal gene therapy. J Inherit Metab Dis 2024;47:192-210. [PMID: 37470194 PMCID: PMC10799196 DOI: 10.1002/jimd.12659] [Citation(s) in RCA: 1] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 03/04/2023] [Revised: 07/11/2023] [Accepted: 07/12/2023] [Indexed: 07/21/2023]
25
Wang X, McKillop WM, Dlugi TA, Faber ML, Alvarez-Argote J, Chambers CB, Wilber A, Medin JA. A mass spectrometry assay for detection of endogenous and lentiviral engineered hemoglobin in cultured cells and sickle cell disease mice. J Gene Med 2024;26:e3567. [PMID: 37455676 DOI: 10.1002/jgm.3567] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/07/2023] [Revised: 06/16/2023] [Accepted: 06/26/2023] [Indexed: 07/18/2023]  Open
26
Uchiyama T, Kawai T, Nakabayashi K, Nakazawa Y, Goto F, Okamura K, Nishimura T, Kato K, Watanabe N, Miura A, Yasuda T, Ando Y, Minegishi T, Edasawa K, Shimura M, Akiba Y, Sato-Otsubo A, Mizukami T, Kato M, Akashi K, Nunoi H, Onodera M. Myelodysplasia after clonal hematopoiesis with APOBEC3-mediated CYBB inactivation in retroviral gene therapy for X-CGD. Mol Ther 2023;31:3424-3440. [PMID: 37705244 PMCID: PMC10727956 DOI: 10.1016/j.ymthe.2023.09.004] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/06/2022] [Revised: 09/02/2023] [Accepted: 09/07/2023] [Indexed: 09/15/2023]  Open
27
Ott de Bruin LM, Lankester AC, Staal FJ. Advances in gene therapy for inborn errors of immunity. Curr Opin Allergy Clin Immunol 2023;23:467-477. [PMID: 37846903 PMCID: PMC10621649 DOI: 10.1097/aci.0000000000000952] [Citation(s) in RCA: 13] [Impact Index Per Article: 6.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 10/18/2023]
28
Minskaia E, Galieva A, Egorov AD, Ivanov R, Karabelsky A. Viral Vectors in Gene Replacement Therapy. BIOCHEMISTRY. BIOKHIMIIA 2023;88:2157-2178. [PMID: 38462459 DOI: 10.1134/s0006297923120179] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Subscribe] [Scholar Register] [Received: 04/28/2023] [Revised: 09/29/2023] [Accepted: 10/17/2023] [Indexed: 03/12/2024]
29
Santiago-Frangos A, Henriques WS, Wiegand T, Gauvin CC, Buyukyoruk M, Graham AB, Wilkinson RA, Triem L, Neselu K, Eng ET, Lander GC, Wiedenheft B. Structure reveals why genome folding is necessary for site-specific integration of foreign DNA into CRISPR arrays. Nat Struct Mol Biol 2023;30:1675-1685. [PMID: 37710013 PMCID: PMC10872659 DOI: 10.1038/s41594-023-01097-2] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/22/2023] [Accepted: 08/15/2023] [Indexed: 09/16/2023]
30
Kohn DB, Chen YY, Spencer MJ. Successes and challenges in clinical gene therapy. Gene Ther 2023;30:738-746. [PMID: 37935854 PMCID: PMC10678346 DOI: 10.1038/s41434-023-00390-5] [Citation(s) in RCA: 46] [Impact Index Per Article: 23.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/01/2022] [Revised: 01/20/2023] [Accepted: 02/07/2023] [Indexed: 11/09/2023]
31
Wadbudhe AM, Meshram RJ, Tidke SC. Severe Combined Immunodeficiency (SCID) and Its New Treatment Modalities. Cureus 2023;15:e47759. [PMID: 38022338 PMCID: PMC10676291 DOI: 10.7759/cureus.47759] [Citation(s) in RCA: 2] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/21/2023] [Accepted: 10/26/2023] [Indexed: 12/01/2023]  Open
32
Bueren JA, Auricchio A. Advances and Challenges in the Development of Gene Therapy Medicinal Products for Rare Diseases. Hum Gene Ther 2023;34:763-775. [PMID: 37694572 DOI: 10.1089/hum.2023.152] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 09/12/2023]  Open
33
Righi M, Gannon I, Robson M, Srivastava S, Kokalaki E, Grothier T, Nannini F, Allen C, Bai YV, Sillibourne J, Cordoba S, Thomas S, Pule M. Enhancing CAR T-cell Therapy Using Fab-Based Constitutively Heterodimeric Cytokine Receptors. Cancer Immunol Res 2023;11:1203-1221. [PMID: 37352396 PMCID: PMC10472109 DOI: 10.1158/2326-6066.cir-22-0640] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/10/2022] [Revised: 03/15/2023] [Accepted: 06/21/2023] [Indexed: 06/25/2023]
34
Piel FB, Rees DC, DeBaun MR, Nnodu O, Ranque B, Thompson AA, Ware RE, Abboud MR, Abraham A, Ambrose EE, Andemariam B, Colah R, Colombatti R, Conran N, Costa FF, Cronin RM, de Montalembert M, Elion J, Esrick E, Greenway AL, Idris IM, Issom DZ, Jain D, Jordan LC, Kaplan ZS, King AA, Lloyd-Puryear M, Oppong SA, Sharma A, Sung L, Tshilolo L, Wilkie DJ, Ohene-Frempong K. Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission. Lancet Haematol 2023;10:e633-e686. [PMID: 37451304 PMCID: PMC11459696 DOI: 10.1016/s2352-3026(23)00096-0] [Citation(s) in RCA: 52] [Impact Index Per Article: 26.0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/31/2023] [Revised: 03/31/2023] [Accepted: 04/12/2023] [Indexed: 07/18/2023]
35
Lundstrom K. Viral vectors engineered for gene therapy. INTERNATIONAL REVIEW OF CELL AND MOLECULAR BIOLOGY 2023;379:1-41. [PMID: 37541721 DOI: 10.1016/bs.ircmb.2023.05.005] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 08/06/2023]
36
Arlabosse T, Booth C, Candotti F. Gene Therapy for Inborn Errors of Immunity. THE JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY. IN PRACTICE 2023;11:1592-1601. [PMID: 37084938 DOI: 10.1016/j.jaip.2023.04.001] [Citation(s) in RCA: 12] [Impact Index Per Article: 6.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Subscribe] [Scholar Register] [Received: 02/13/2023] [Revised: 04/06/2023] [Accepted: 04/07/2023] [Indexed: 04/23/2023]
37
Ferrari S, Valeri E, Conti A, Scala S, Aprile A, Di Micco R, Kajaste-Rudnitski A, Montini E, Ferrari G, Aiuti A, Naldini L. Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy. Cell Stem Cell 2023;30:549-570. [PMID: 37146580 DOI: 10.1016/j.stem.2023.04.014] [Citation(s) in RCA: 31] [Impact Index Per Article: 15.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/01/2023] [Revised: 03/31/2023] [Accepted: 04/12/2023] [Indexed: 05/07/2023]
38
Slatter M, Lum SH. Personalized hematopoietic stem cell transplantation for inborn errors of immunity. Front Immunol 2023;14:1162605. [PMID: 37090739 PMCID: PMC10113466 DOI: 10.3389/fimmu.2023.1162605] [Citation(s) in RCA: 3] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/09/2023] [Accepted: 03/20/2023] [Indexed: 04/08/2023]  Open
39
Ugalde L, Fañanas S, Torres R, Quintana-Bustamante O, Río P. CRISPR/Cas9-mediated gene editing. A promising strategy in hematological disorders. Cytotherapy 2023;25:277-285. [PMID: 36610813 DOI: 10.1016/j.jcyt.2022.11.014] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/15/2021] [Revised: 11/09/2022] [Accepted: 11/30/2022] [Indexed: 01/07/2023]
40
Hara H, Munkh-Erdene N, Byambaa S, Hanazono Y. Nonviral Ex Vivo Genome Editing in Mouse Bona Fide Hematopoietic Stem Cells with CRISPR/Cas9. Methods Mol Biol 2023;2637:213-221. [PMID: 36773149 DOI: 10.1007/978-1-0716-3016-7_16] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 02/12/2023]
41
A Review of Different Types of Liposomes and Their Advancements as a Form of Gene Therapy Treatment for Breast Cancer. Molecules 2023;28:molecules28031498. [PMID: 36771161 PMCID: PMC9920768 DOI: 10.3390/molecules28031498] [Citation(s) in RCA: 39] [Impact Index Per Article: 19.5] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/10/2022] [Revised: 11/30/2022] [Accepted: 12/04/2022] [Indexed: 02/09/2023]  Open
42
Chen MJ, Gatignol A, Scarborough RJ. The discovery and development of RNA-based therapies for treatment of HIV-1 infection. Expert Opin Drug Discov 2023;18:163-179. [PMID: 36004505 DOI: 10.1080/17460441.2022.2117296] [Citation(s) in RCA: 4] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 02/07/2023]
43
Mudde A, Booth C. Gene therapy for inborn error of immunity - current status and future perspectives. Curr Opin Allergy Clin Immunol 2023;23:51-62. [PMID: 36539381 DOI: 10.1097/aci.0000000000000876] [Citation(s) in RCA: 8] [Impact Index Per Article: 4.0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/24/2022]
44
Progress of Endogenous and Exogenous Nanoparticles for Cancer Therapy and Diagnostics. Genes (Basel) 2023;14:genes14020259. [PMID: 36833186 PMCID: PMC9957423 DOI: 10.3390/genes14020259] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/29/2022] [Revised: 01/12/2023] [Accepted: 01/17/2023] [Indexed: 01/20/2023]  Open
45
Buffa V, Alvarez Vargas JR, Galy A, Spinozzi S, Rocca CJ. Hematopoietic stem and progenitors cells gene editing: Beyond blood disorders. Front Genome Ed 2023;4:997142. [PMID: 36698790 PMCID: PMC9868335 DOI: 10.3389/fgeed.2022.997142] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 07/18/2022] [Accepted: 12/19/2022] [Indexed: 01/10/2023]  Open
46
Brault J, Liu T, Liu S, Lawson A, Choi U, Kozhushko N, Bzhilyanskaya V, Pavel-Dinu M, Meis RJ, Eckhaus MA, Burkett SS, Bosticardo M, Kleinstiver BP, Notarangelo LD, Lazzarotto CR, Tsai SQ, Wu X, Dahl GA, Porteus MH, Malech HL, De Ravin SS. CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells. Front Immunol 2023;13:1067417. [PMID: 36685559 PMCID: PMC9846165 DOI: 10.3389/fimmu.2022.1067417] [Citation(s) in RCA: 13] [Impact Index Per Article: 6.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/11/2022] [Accepted: 12/06/2022] [Indexed: 01/05/2023]  Open
47
Lundstrom K. Gene Therapy Cargoes Based on Viral Vector Delivery. Curr Gene Ther 2023;23:111-134. [PMID: 36154608 DOI: 10.2174/1566523222666220921112753] [Citation(s) in RCA: 5] [Impact Index Per Article: 2.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/08/2022] [Revised: 07/13/2022] [Accepted: 08/05/2022] [Indexed: 11/22/2022]
48
Solomon M, Liang C. Pseudotyped Viruses for Retroviruses. ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY 2023;1407:61-84. [PMID: 36920692 DOI: 10.1007/978-981-99-0113-5_4] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 03/16/2023]
49
van Heuvel Y, Schatz S, Hein M, Dogra T, Kazenmaier D, Tschorn N, Genzel Y, Stitz J. Novel suspension retroviral packaging cells generated by transposition using transposase encoding mRNA advance vector yields and enable production in bioreactors. Front Bioeng Biotechnol 2023;11:1076524. [PMID: 37082212 PMCID: PMC10112512 DOI: 10.3389/fbioe.2023.1076524] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/21/2022] [Accepted: 03/24/2023] [Indexed: 04/22/2023]  Open
50
Tsujimoto H, Osafune K. Current status and future directions of clinical applications using iPS cells-focus on Japan. FEBS J 2022;289:7274-7291. [PMID: 34407307 DOI: 10.1111/febs.16162] [Citation(s) in RCA: 11] [Impact Index Per Article: 3.7] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/22/2021] [Revised: 08/04/2021] [Accepted: 08/17/2021] [Indexed: 01/13/2023]
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