Below are the most recent publications written about "Genetic Therapy" by people in Profiles.
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Pires Ferreira D, Gruntman AM, Flotte TR. Gene therapy for alpha-1 antitrypsin deficiency: an update. Expert Opin Biol Ther. 2023 Mar; 23(3):283-291.
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McDonald CL, Qasba P, Anderson DG, Bao G, Colvin RA, Kohn DB, Malik P, Mitchell MJ, Pu WT, Rawlings DJ, Williams DA, Flotte TR. Future Directions and Resource Needs for National Heart, Lung, and Blood Institute (NHLBI) Gene Therapy Research: A Report of an NHLBI Workshop. Hum Gene Ther. 2023 02; 34(3-4):83-89.
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Yang YS, Kim JM, Xie J, Chaugule S, Lin C, Ma H, Hsiao E, Hong J, Chun H, Shore EM, Kaplan FS, Gao G, Shim JH. Suppression of heterotopic ossification in fibrodysplasia ossificans progressiva using AAV gene delivery. Nat Commun. 2022 10 19; 13(1):6175.
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Gonzalez TJ, Simon KE, Blondel LO, Fanous MM, Roger AL, Maysonet MS, Devlin GW, Smith TJ, Oh DK, Havlik LP, Castellanos Rivera RM, Piedrahita JA, ElMallah MK, Gersbach CA, Asokan A. Cross-species evolution of a highly potent AAV variant for therapeutic gene transfer and genome editing. Nat Commun. 2022 10 10; 13(1):5947.
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Oh WT, Yang YS, Xie J, Ma H, Kim JM, Park KH, Oh DS, Park-Min KH, Greenblatt MB, Gao G, Shim JH. WNT-modulating gene silencers as a gene therapy for osteoporosis, bone fracture, and critical-sized bone defects. Mol Ther. 2023 02 01; 31(2):435-453.
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Cheng SY, Punzo C. Update on Viral Gene Therapy Clinical Trials for Retinal Diseases. Hum Gene Ther. 2022 09; 33(17-18):865-878.
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McElroy A, Sena-Esteves M, Arjomandnejad M, Keeler AM, Gray-Edwards HL. Redosing Adeno-Associated Virus Gene Therapy to the Central Nervous System. Hum Gene Ther. 2022 09; 33(17-18):889-892.
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Flotte TR. ESGCT 2022: The New Normal for the Gene Therapy Community. Hum Gene Ther. 2022 09; 33(17-18):841.
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Pavan-Guimaraes J, Martins PN. Modifying organs with gene therapy and gene modulation in the age of machine perfusion. Curr Opin Organ Transplant. 2022 10 01; 27(5):474-480.
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Eekhoff EMW, de Ruiter RD, Smilde BJ, Schoenmaker T, de Vries TJ, Netelenbos C, Hsiao EC, Scott C, Haga N, Grunwald Z, De Cunto CL, di Rocco M, Delai PLR, Diecidue RJ, Madhuri V, Cho TJ, Morhart R, Friedman CS, Zasloff M, Pals G, Shim JH, Gao G, Kaplan F, Pignolo RJ, Micha D. Gene Therapy for Fibrodysplasia Ossificans Progressiva: Feasibility and Obstacles. Hum Gene Ther. 2022 08; 33(15-16):782-788.