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For: Hacein-Bey-Abina S, Hauer J, Lim A, Picard C, Wang GP, Berry CC, Martinache C, Rieux-Laucat F, Latour S, Belohradsky BH, Leiva L, Sorensen R, Debré M, Casanova JL, Blanche S, Durandy A, Bushman FD, Fischer A, Cavazzana-Calvo M. Efficacy of gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2010;363:355-64. [PMID: 20660403 PMCID: PMC2957288 DOI: 10.1056/nejmoa1000164] [Citation(s) in RCA: 419] [Impact Index Per Article: 27.9] [Reference Citation Analysis] [What about the content of this article? (0)] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 12/11/2022]
Number Cited by Other Article(s)
1
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
2
Stevens CS, Carmichael JC, Watkinson R, Kowdle S, Reis RA, Hamane K, Jang J, Park A, Pernet O, Khamaikawin W, Hong P, Thibault P, Gowlikar A, An DS, Lee B. A temperature-sensitive and less immunogenic Sendai virus for efficient gene editing. J Virol 2024;98:e0083224. [PMID: 39494910 PMCID: PMC11650993 DOI: 10.1128/jvi.00832-24] [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: 05/09/2024] [Accepted: 10/10/2024] [Indexed: 11/05/2024]  Open
3
Karimi-Sani I, Molavi Z, Naderi S, Mirmajidi SH, Zare I, Naeimzadeh Y, Mansouri A, Tajbakhsh A, Savardashtaki A, Sahebkar A. Personalized mRNA vaccines in glioblastoma therapy: from rational design to clinical trials. J Nanobiotechnology 2024;22:601. [PMID: 39367418 PMCID: PMC11453023 DOI: 10.1186/s12951-024-02882-x] [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: 06/03/2024] [Accepted: 09/26/2024] [Indexed: 10/06/2024]  Open
4
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
5
Graham JP, Castro JG, Werba LC, Fardone LC, Francis KP, Ramamurthi A, Layden M, McCarthy HO, Gonzalez-Fernandez T. Versatile Cell Penetrating Peptide for Multimodal CRISPR Gene Editing in Primary Stem Cells. BIORXIV : THE PREPRINT SERVER FOR BIOLOGY 2024:2024.09.23.614499. [PMID: 39386541 PMCID: PMC11463527 DOI: 10.1101/2024.09.23.614499] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Download PDF] [Subscribe] [Scholar Register] [Indexed: 10/12/2024]
6
Afzal SY, MacDougall MS, McGhee SA. Immunodeficiency: Gene therapy for primary immune deficiency. Allergy Asthma Proc 2024;45:384-388. [PMID: 39294901 DOI: 10.2500/aap.2024.45.240054] [Citation(s) in RCA: 1] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 09/21/2024]
7
Rozenbaum M, Fluss R, Marcu-Malina V, Sarouk I, Meir A, Elitzur S, Zinger T, Jacob-Hirsch J, Saar EG, Rechavi G, Jacoby E. Genotoxicity Associated with Retroviral CAR Transduction of ATM-Deficient T Cells. Blood Cancer Discov 2024;5:267-275. [PMID: 38747501 PMCID: PMC11215369 DOI: 10.1158/2643-3230.bcd-23-0268] [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: 12/20/2023] [Revised: 02/26/2024] [Accepted: 05/08/2024] [Indexed: 07/02/2024]  Open
8
McIvor RS, Eaton EJ, Webber BR, Moriarity BS. Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1. Mol Ther 2024;32:1606-1608. [PMID: 38781958 PMCID: PMC11184398 DOI: 10.1016/j.ymthe.2024.05.026] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 05/03/2024] [Revised: 05/10/2024] [Accepted: 05/10/2024] [Indexed: 05/25/2024]  Open
9
Stevens CS, Carmichael J, Watkinson R, Kowdle S, Reis RA, Hamane K, Jang J, Park A, Pernet O, Khamaikawin W, Hong P, Thibault P, Gowlikar A, An DS, Lee B. A temperature-sensitive and interferon-silent Sendai virus vector for CRISPR-Cas9 delivery and gene editing in primary human cells. BIORXIV : THE PREPRINT SERVER FOR BIOLOGY 2024:2024.05.03.592383. [PMID: 38746439 PMCID: PMC11092779 DOI: 10.1101/2024.05.03.592383] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 05/16/2024]
10
Lemmens M, Dorsheimer L, Zeller A, Dietz-Baum Y. Non-clinical safety assessment of novel drug modalities: Genome safety perspectives on viral-, nuclease- and nucleotide-based gene therapies. MUTATION RESEARCH. GENETIC TOXICOLOGY AND ENVIRONMENTAL MUTAGENESIS 2024;896:503767. [PMID: 38821669 DOI: 10.1016/j.mrgentox.2024.503767] [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: 12/18/2023] [Revised: 04/08/2024] [Accepted: 05/13/2024] [Indexed: 06/02/2024]
11
Cesana D, Cicalese MP, Calabria A, Merli P, Caruso R, Volpin M, Rudilosso L, Migliavacca M, Barzaghi F, Fossati C, Gazzo F, Pizzi S, Ciolfi A, Bruselles A, Tucci F, Spinozzi G, Pais G, Benedicenti F, Barcella M, Merelli I, Gallina P, Giannelli S, Dionisio F, Scala S, Casiraghi M, Strocchio L, Vinti L, Pacillo L, Draghi E, Cesana M, Riccardo S, Colantuono C, Six E, Cavazzana M, Carlucci F, Schmidt M, Cancrini C, Ciceri F, Vago L, Cacchiarelli D, Gentner B, Naldini L, Tartaglia M, Montini E, Locatelli F, Aiuti A. A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID. Nat Commun 2024;15:3662. [PMID: 38688902 PMCID: PMC11061298 DOI: 10.1038/s41467-024-47866-5] [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: 10/13/2023] [Accepted: 04/10/2024] [Indexed: 05/02/2024]  Open
12
Rossi A, Brunetti-Pierri N. Gene therapies for mucopolysaccharidoses. J Inherit Metab Dis 2024;47:135-144. [PMID: 37204267 DOI: 10.1002/jimd.12626] [Citation(s) in RCA: 9] [Impact Index Per Article: 9.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 01/12/2023] [Revised: 04/27/2023] [Accepted: 05/15/2023] [Indexed: 05/20/2023]
13
Liu Y, Karlsson S. Perspectives of current understanding and therapeutics of Diamond-Blackfan anemia. Leukemia 2024;38:1-9. [PMID: 37973818 PMCID: PMC10776401 DOI: 10.1038/s41375-023-02082-w] [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: 08/18/2023] [Revised: 10/20/2023] [Accepted: 11/06/2023] [Indexed: 11/19/2023]
14
Karpov DS, Sosnovtseva AO, Pylina SV, Bastrich AN, Petrova DA, Kovalev MA, Shuvalova AI, Eremkina AK, Mokrysheva NG. Challenges of CRISPR/Cas-Based Cell Therapy for Type 1 Diabetes: How Not to Engineer a "Trojan Horse". Int J Mol Sci 2023;24:17320. [PMID: 38139149 PMCID: PMC10743607 DOI: 10.3390/ijms242417320] [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: 11/03/2023] [Revised: 12/04/2023] [Accepted: 12/07/2023] [Indexed: 12/24/2023]  Open
15
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
16
Voit RA, Corey SJ. Gene therapy for congenital marrow failure syndromes - no longer grasping at straws? Haematologica 2023;108:2880-2882. [PMID: 37317900 PMCID: PMC10620564 DOI: 10.3324/haematol.2023.283462] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/01/2023] [Accepted: 06/06/2023] [Indexed: 06/16/2023]  Open
17
Riller Q, Fourgeaud J, Bruneau J, De Ravin SS, Smith G, Fusaro M, Meriem S, Magerus A, Luka M, Abdessalem G, Lhermitte L, Jamet A, Six E, Magnani A, Castelle M, Lévy R, Lecuit MM, Fournier B, Winter S, Semeraro M, Pinto G, Abid H, Mahlaoui N, Cheikh N, Florkin B, Frange P, Jeziorski E, Suarez F, Sarrot-Reynauld F, Nouar D, Debray D, Lacaille F, Picard C, Pérot P, Regnault B, Da Rocha N, de Cevins C, Delage L, Pérot BP, Vinit A, Carbone F, Brunaud C, Marchais M, Stolzenberg MC, Asnafi V, Molina T, Rieux-Laucat F, Notarangelo LD, Pittaluga S, Jais JP, Moshous D, Blanche S, Malech H, Eloit M, Cavazzana M, Fischer A, Ménager MM, Neven B. Late-onset enteric virus infection associated with hepatitis (EVAH) in transplanted SCID patients. J Allergy Clin Immunol 2023;151:1634-1645. [PMID: 36638922 PMCID: PMC10336473 DOI: 10.1016/j.jaci.2022.12.822] [Citation(s) in RCA: 3] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/16/2022] [Revised: 12/14/2022] [Accepted: 12/21/2022] [Indexed: 01/12/2023]
18
Zhou H, He Y, Xiong W, Jing S, Duan X, Huang Z, Nahal GS, Peng Y, Li M, Zhu Y, Ye Q. MSC based gene delivery methods and strategies improve the therapeutic efficacy of neurological diseases. Bioact Mater 2023;23:409-437. [PMCID: PMC9713256 DOI: 10.1016/j.bioactmat.2022.11.007] [Citation(s) in RCA: 9] [Impact Index Per Article: 4.5] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 09/29/2022] [Revised: 11/08/2022] [Accepted: 11/13/2022] [Indexed: 12/05/2022]  Open
19
Allen D, Kalter N, Rosenberg M, Hendel A. Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood Disorders. Pharmaceutics 2023;15:1329. [PMID: 37242571 PMCID: PMC10220672 DOI: 10.3390/pharmaceutics15051329] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/09/2023] [Revised: 04/13/2023] [Accepted: 04/19/2023] [Indexed: 05/28/2023]  Open
20
Castiello MC, Ferrari S, Villa A. Correcting inborn errors of immunity: From viral mediated gene addition to gene editing. Semin Immunol 2023;66:101731. [PMID: 36863140 PMCID: PMC10109147 DOI: 10.1016/j.smim.2023.101731] [Citation(s) in RCA: 2] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 10/03/2022] [Revised: 01/25/2023] [Accepted: 02/14/2023] [Indexed: 03/04/2023]
21
Zhao Z, Grégoire C, Oliveira B, Chung K, Melenhorst JJ. Challenges and opportunities of CAR T cell therapies for CLL. Semin Hematol 2023;60:25-33. [PMID: 37080707 DOI: 10.1053/j.seminhematol.2023.01.002] [Citation(s) in RCA: 3] [Impact Index Per Article: 1.5] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 01/03/2023] [Revised: 01/10/2023] [Accepted: 01/19/2023] [Indexed: 02/10/2023]
22
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: 5] [Impact Index Per Article: 2.5] [Reference Citation Analysis] [Abstract] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/24/2022]
23
Koh AL, Jamuar SS. Therapeutics in paediatric genetic diseases: Current and future landscape. Singapore Med J 2023;64:7-16. [PMID: 36722512 PMCID: PMC9979794 DOI: 10.4103/singaporemedj.smj-2021-376] [Citation(s) in RCA: 2] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/20/2023]
24
Kuo CY, Kohn DB. Gene Therapy for Primary Immune Deficiency Diseases. Clin Immunol 2023. [DOI: 10.1016/b978-0-7020-8165-1.00091-5] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 04/09/2023]
25
Chakraborty A, Dharmaraj S, Truong N, Pearson RM. Excipient-Free Ionizable Polyester Nanoparticles for Lung-Selective and Innate Immune Cell Plasmid DNA and mRNA Transfection. ACS APPLIED MATERIALS & INTERFACES 2022;14:56440-56453. [PMID: 36525379 PMCID: PMC9872050 DOI: 10.1021/acsami.2c14424] [Citation(s) in RCA: 6] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 06/17/2023]
26
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]
27
Hernandez JD, Hsieh EW. A great disturbance in the force: IL-2 receptor defects disrupt immune homeostasis. Curr Opin Pediatr 2022;34:580-588. [PMID: 36165614 PMCID: PMC9633542 DOI: 10.1097/mop.0000000000001181] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Indexed: 01/13/2023]
28
Sharma A, Farnia S, Otegbeye F, Rinkle A, Shah J, Shah NN, Gill S, Maus MV. Nomenclature for Cellular and Genetic Therapies: A Need for Standardization. Transplant Cell Ther 2022;28:795-801. [PMID: 36058548 PMCID: PMC9729423 DOI: 10.1016/j.jtct.2022.08.029] [Citation(s) in RCA: 3] [Impact Index Per Article: 1.0] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 08/24/2022] [Revised: 08/25/2022] [Accepted: 08/26/2022] [Indexed: 12/24/2022]
29
Fischer A. Gene therapy for inborn errors of immunity: past, present and future. Nat Rev Immunol 2022:10.1038/s41577-022-00800-6. [DOI: 10.1038/s41577-022-00800-6] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 10/26/2022] [Indexed: 11/27/2022]
30
Shabaneh TB, Moffett HF, Stull SM, Derezes T, Tait LJ, Park S, Riddell SR, Lajoie MJ. Safety switch optimization enhances antibody-mediated elimination of CAR T cells. FRONTIERS IN MOLECULAR MEDICINE 2022;2:1026474. [PMID: 39086975 PMCID: PMC11285702 DOI: 10.3389/fmmed.2022.1026474] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Figures] [Subscribe] [Scholar Register] [Received: 08/23/2022] [Accepted: 09/27/2022] [Indexed: 08/02/2024]
31
Falcon C, Smith L, Al-Obaidi M, Abu Zaanona M, Purvis K, Minagawa K, Athar M, Salzman D, Bhatia R, Goldman F, Di Stasi A. Combinatorial suicide gene strategies for the safety of cell therapies. Front Immunol 2022;13:975233. [PMID: 36189285 PMCID: PMC9515659 DOI: 10.3389/fimmu.2022.975233] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/22/2022] [Accepted: 08/29/2022] [Indexed: 11/13/2022]  Open
32
Palamenghi M, De Luca M, De Rosa L. The steep uphill path leading to ex vivo gene therapy for genodermatoses. Am J Physiol Cell Physiol 2022;323:C896-C906. [PMID: 35912986 DOI: 10.1152/ajpcell.00117.2022] [Citation(s) in RCA: 6] [Impact Index Per Article: 2.0] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 11/22/2022]
33
Chetty K, Houghton BC, Booth C. Gene Therapy for Inborn Errors of Immunity. Hematol Oncol Clin North Am 2022;36:813-827. [DOI: 10.1016/j.hoc.2022.03.010] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 01/03/2023]
34
Mesenchymal stem cells: A living carrier for active tumor-targeted delivery. Adv Drug Deliv Rev 2022;185:114300. [PMID: 35447165 DOI: 10.1016/j.addr.2022.114300] [Citation(s) in RCA: 23] [Impact Index Per Article: 7.7] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/26/2021] [Revised: 03/22/2022] [Accepted: 04/12/2022] [Indexed: 12/16/2022]
35
Konturek-Ciesla A, Bryder D. Stem Cells, Hematopoiesis and Lineage Tracing: Transplantation-Centric Views and Beyond. Front Cell Dev Biol 2022;10:903528. [PMID: 35573680 PMCID: PMC9091331 DOI: 10.3389/fcell.2022.903528] [Citation(s) in RCA: 7] [Impact Index Per Article: 2.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 03/24/2022] [Accepted: 04/12/2022] [Indexed: 12/26/2022]  Open
36
Ravendran S, Hernández SS, König S, Bak RO. CRISPR/Cas-Based Gene Editing Strategies for DOCK8 Immunodeficiency Syndrome. Front Genome Ed 2022;4:793010. [PMID: 35373187 PMCID: PMC8969908 DOI: 10.3389/fgeed.2022.793010] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.7] [Reference Citation Analysis] [Abstract] [Key Words] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/11/2021] [Accepted: 02/14/2022] [Indexed: 12/17/2022]  Open
37
Tucci F, Galimberti S, Naldini L, Valsecchi MG, Aiuti A. A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders. Nat Commun 2022;13:1315. [PMID: 35288539 PMCID: PMC8921234 DOI: 10.1038/s41467-022-28762-2] [Citation(s) in RCA: 77] [Impact Index Per Article: 25.7] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 06/06/2021] [Accepted: 02/08/2022] [Indexed: 12/12/2022]  Open
38
Sayed N, Allawadhi P, Khurana A, Singh V, Navik U, Pasumarthi SK, Khurana I, Banothu AK, Weiskirchen R, Bharani KK. Gene therapy: Comprehensive overview and therapeutic applications. Life Sci 2022;294:120375. [PMID: 35123997 DOI: 10.1016/j.lfs.2022.120375] [Citation(s) in RCA: 112] [Impact Index Per Article: 37.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 12/17/2021] [Revised: 01/24/2022] [Accepted: 01/31/2022] [Indexed: 02/07/2023]
39
Regenerative Medicine Application of Mesenchymal Stem Cells. ADVANCES IN EXPERIMENTAL MEDICINE AND BIOLOGY 2022;1387:25-42. [DOI: 10.1007/5584_2022_713] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Track Full Text] [Subscribe] [Scholar Register] [Indexed: 12/09/2022]
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Development and clinical translation of ex vivo gene therapy. Comput Struct Biotechnol J 2022;20:2986-3003. [PMID: 35782737 PMCID: PMC9218169 DOI: 10.1016/j.csbj.2022.06.015] [Citation(s) in RCA: 0] [Impact Index Per Article: 0] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 02/08/2022] [Revised: 06/07/2022] [Accepted: 06/07/2022] [Indexed: 11/27/2022]  Open
41
Iggo R. Lentiviral Transduction of Mammary Epithelial Cells. Methods Mol Biol 2022;2471:159-183. [PMID: 35175596 DOI: 10.1007/978-1-0716-2193-6_8] [Citation(s) in RCA: 4] [Impact Index Per Article: 1.3] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 06/14/2023]
42
A Nanoparticle-Conjugated Anti-TBK1 siRNA Induces Autophagy-Related Apoptosis and Enhances cGAS-STING Pathway in GBM Cells. EVIDENCE-BASED COMPLEMENTARY AND ALTERNATIVE MEDICINE 2021;2021:6521953. [PMID: 34931127 PMCID: PMC8684524 DOI: 10.1155/2021/6521953] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Download PDF] [Figures] [Subscribe] [Scholar Register] [Received: 07/07/2021] [Accepted: 11/05/2021] [Indexed: 11/30/2022]
43
Genetic Modification of Mesenchymal Stem Cells for Neurological Disease Therapy: What Effects Does it Have on Phenotype/Cell Behavior, Determining Their Effectiveness? Mol Diagn Ther 2021;24:683-702. [PMID: 32926348 DOI: 10.1007/s40291-020-00491-6] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Indexed: 12/16/2022]
44
Morgan MA, Galla M, Grez M, Fehse B, Schambach A. Retroviral gene therapy in Germany with a view on previous experience and future perspectives. Gene Ther 2021;28:494-512. [PMID: 33753908 PMCID: PMC8455336 DOI: 10.1038/s41434-021-00237-x] [Citation(s) in RCA: 15] [Impact Index Per Article: 3.8] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 10/02/2020] [Revised: 01/13/2021] [Accepted: 02/01/2021] [Indexed: 02/01/2023]
45
Bode D, Cull AH, Rubio-Lara JA, Kent DG. Exploiting Single-Cell Tools in Gene and Cell Therapy. Front Immunol 2021;12:702636. [PMID: 34322133 PMCID: PMC8312222 DOI: 10.3389/fimmu.2021.702636] [Citation(s) in RCA: 22] [Impact Index Per Article: 5.5] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Grants] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Received: 04/29/2021] [Accepted: 06/28/2021] [Indexed: 12/12/2022]  Open
46
Baeten P, Van Zeebroeck L, Kleinewietfeld M, Hellings N, Broux B. Improving the Efficacy of Regulatory T Cell Therapy. Clin Rev Allergy Immunol 2021;62:363-381. [PMID: 34224053 PMCID: PMC8256646 DOI: 10.1007/s12016-021-08866-1] [Citation(s) in RCA: 25] [Impact Index Per Article: 6.3] [Reference Citation Analysis] [Abstract] [Key Words] [Track Full Text] [Download PDF] [Figures] [Journal Information] [Subscribe] [Scholar Register] [Accepted: 05/31/2021] [Indexed: 12/11/2022]
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Ramos CV, Martins VC. Cell competition in hematopoietic cells: Quality control in homeostasis and its role in leukemia. Dev Biol 2021;475:1-9. [DOI: 10.1016/j.ydbio.2021.02.013] [Citation(s) in RCA: 2] [Impact Index Per Article: 0.5] [Reference Citation Analysis] [Track Full Text] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/23/2020] [Revised: 02/19/2021] [Accepted: 02/23/2021] [Indexed: 12/24/2022]
48
Galanopoulos M, Doukatas A, Gkeros F, Viazis N, Liatsos C. Room for improvement in the treatment of pancreatic cancer: Novel opportunities from gene targeted therapy. World J Gastroenterol 2021;27:3568-3580. [PMID: 34239270 PMCID: PMC8240062 DOI: 10.3748/wjg.v27.i24.3568] [Citation(s) in RCA: 3] [Impact Index Per Article: 0.8] [Reference Citation Analysis] [Abstract] [Key Words] [MESH Headings] [Track Full Text] [Download PDF] [Journal Information] [Submit a Manuscript] [Subscribe] [Scholar Register] [Received: 02/16/2021] [Revised: 04/11/2021] [Accepted: 05/22/2021] [Indexed: 02/06/2023]  Open
49
Retrieval of vector integration sites from cell-free DNA. Nat Med 2021;27:1458-1470. [PMID: 34140705 DOI: 10.1038/s41591-021-01389-4] [Citation(s) in RCA: 24] [Impact Index Per Article: 6.0] [Reference Citation Analysis] [Abstract] [Journal Information] [Subscribe] [Scholar Register] [Received: 11/11/2020] [Accepted: 05/07/2021] [Indexed: 12/23/2022]
50
Sipe CJ, Claudio Vázquez PN, Skeate JG, McIvor RS, Moriarity BS. Targeted genome editing for the correction or alleviation of primary Immunodeficiencies. PROGRESS IN MOLECULAR BIOLOGY AND TRANSLATIONAL SCIENCE 2021;182:111-151. [PMID: 34175040 DOI: 10.1016/bs.pmbts.2021.03.001] [Citation(s) in RCA: 1] [Impact Index Per Article: 0.3] [Reference Citation Analysis] [Abstract] [Key Words] [Subscribe] [Scholar Register] [Indexed: 12/26/2022]
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