Below are the most recent publications written about "Genetic Therapy" by people in Profiles.
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Celik B, Leal AF, Tomatsu S. Potential Targeting Mechanisms for Bone-Directed Therapies. Int J Mol Sci. 2024 Jul 30; 25(15).
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Baratta M, Jian W, Hengel S, Kaur S, Cunliffe J, Boer J, Hughes N, Kar S, Kellie J, Kim YJ, Lassman M, Mehl J, Morgan L, Palandra J, Sarvaiya H, Zeng J, Zheng N, Wang J, Yuan L, Ji A, Kochansky C, Tao L, Huang Y, Maes E, Barbero L, Contrepois K, Ferrari L, Fu Y, Johnson J, Jones B, Kansal M, Lu Y, Post N, Shen HH, Xue YY, Zhang YC, Biswas G, Cho SJ, Edmison A, Benson K, Abberley L, Azadeh M, Francis J, Garofolo F, Gupta S, Ivanova ID, Ishii-Watabe A, Karnik S, Kassim S, Kavetska O, Keller S, Kossary E, Li W, McCush F, Mendes DN, Abhari MR, Scheibner K, Sikorski T, Staack RF, Tabler E, Tang H, Wan K, Wang YM, Whale E, Yang L, Zimmer J, Bandukwala A, Du X, Kholmanskikh O, Gijsel SK, Wadhwa M, Xu J, Buoninfante A, Cludts I, Diebold S, Maxfield K, Mayer C, Pedras-Vasconcelos J, Abhari MR, Shubow S, Tanaka Y, Tounekti O, Verthelyi D, Wagner L. 2023 White Paper on Recent Issues in Bioanalysis: Deuterated Drugs; LNP; Tumor/FFPE Biopsy; Targeted Proteomics; Small Molecule Covalent Inhibitors; Chiral Bioanalysis; Remote Regulatory Assessments; Sample Reconciliation/Chain of Custody (PART?1A - Recommendations on Mass Spectrometry, Chromatography, Sample Preparation Latest Developments, Challenges, and Solutions and BMV/Regulated Bioanalysis PART?1B - Regulatory Agencies' Inputs on Regulated Bioanalysis/BMV, Biomarkers/IVD/CDx/BAV, Immunogenicity, Gene & Cell Therapy and Vaccine). Bioanalysis. 2024; 16(9):307-364.
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Lau CH, Rouhani MJ, Maughan EF, Orr JC, Kolluri KK, Pearce DR, Haughey EK, Sutton L, Flatau S, Balboa PL, Bageta ML, O'Callaghan C, Smith CM, Janes SM, Hewitt R, Petrof G, Martinez AE, McGrath JA, Butler CR, Hynds RE. Lentiviral expression of wild-type LAMA3A restores cell adhesion in airway basal cells from children with epidermolysis bullosa. Mol Ther. 2024 May 01; 32(5):1497-1509.
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Leal AF, Herreno-Pach?n AM, Benincore-Fl?rez E, Karunathilaka A, Tomatsu S. Current Strategies for Increasing Knock-In Efficiency in CRISPR/Cas9-Based Approaches. Int J Mol Sci. 2024 Feb 20; 25(5).
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Doolan BJ, McGrath JA, Mellerio JE. Beremagene geperpavec (B-VEC) gene therapy for the treatment of cutaneous wounds in patients with dystrophic epidermolysis bullosa: a critically appraised research paper. Br J Dermatol. 2024 Feb 16; 190(3):340-342.
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Katz MG, Hadas Y, Vincek A, Freage-Kahn L, Shtraizent N, Madjarov JM, Pastuszko P, Eliyahu E. Acid ceramidase gene therapy ameliorates pulmonary arterial hypertension with right heart dysfunction. Respir Res. 2023 Aug 11; 24(1):197.
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Leal AF, Fnu N, Benincore-Fl?rez E, Herre?o-Pach?n AM, Echeverri-Pe?a OY, Alm?ciga-D?az CJ, Tomatsu S. The landscape of CRISPR/Cas9 for inborn errors of metabolism. Mol Genet Metab. 2023 01; 138(1):106968.
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Piechnik M, Amendum PC, Sawamoto K, Stapleton M, Khan S, Fnu N, ?lvarez V, Pachon AMH, Danos O, Bruder JT, Karumuthil-Melethil S, Tomatsu S. Sex Difference Leads to Differential Gene Expression Patterns and Therapeutic Efficacy in Mucopolysaccharidosis IVA Murine Model Receiving AAV8 Gene Therapy. Int J Mol Sci. 2022 Oct 21; 23(20).
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Katz MG, Hadas Y, Vincek AS, Shtraizent N, Schadt E, Eliyahu E. Cardiac Targeted Adeno-Associated Virus Injection in Rats. Methods Mol Biol. 2022; 2573:135-145.
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Katz MG, Hadas Y, Vincek AS, Shtraizent N, Gordon HP, Pastuszko P, Schadt E, Eliyahu E. Surgical Methods for Cardiac Gene Delivery in Large Animals. Methods Mol Biol. 2022; 2573:189-203.