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438 results

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Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations.
Yoon JK, Schindler JW, Loperfido M, Baricordi C, DeAndrade MP, Jacobs ME, Treleaven C, Plasschaert RN, Yan A, Barese CN, Dogan Y, Chen VP, Fiorini C, Hull F, Barbarossa L, Unnisa Z, Ivanov D, Kutner RH, Guda S, Oborski C, Maiwald T, Michaud V, Rothe M, Schambach A, Pfeifer R, Mason C, Biasco L, van Til NP. Yoon JK, et al. Among authors: rothe m. Mol Ther. 2024 Sep 17:S1525-0016(24)00606-3. doi: 10.1016/j.ymthe.2024.09.024. Online ahead of print. Mol Ther. 2024. PMID: 39295144
Pharmacological and pre-clinical safety profile of rSIV.F/HN, a hybrid lentiviral vector for cystic fibrosis gene therapy.
Moiseenko A, Sinadinos A, Sergijenko A, Pineault K, Saleh A, Nekola K, Strang N, Eleftheraki A, Boyd AC, Davies JC, Gill DR, Hyde SC, McLachlan G, Rath T, Rothe M, Schambach A, Hobbie S, Schuler M, Maier U, Thomas MJ, Mennerich D, Schmidt M, Griesenbach U, Alton EWFW, Kreuz S. Moiseenko A, et al. Among authors: rothe m. Eur Respir J. 2024 Aug 22:2301683. doi: 10.1183/13993003.01683-2023. Online ahead of print. Eur Respir J. 2024. PMID: 39174284
Improving the Assessment of Risk Factors Relevant to Potential Carcinogenicity of Gene Therapies: A Consensus Article.
Klapwijk JC, Del Rio Espinola A, Libertini S, Collin P, Fellows MD, Jobling S, Lynch AM, Martus H, Vickers C, Zeller A, Biasco L, Brugman MH, Bushmann FD, Cathomen T, Ertl HCJ, Gabriel R, Gao G, Jadlowsky JK, Kimber I, Lanz TA, Levine BL, Micklethwaite KP, Onodera M, Pizzurro DM, Reed S, Rothe M, Sabatino DE, Salk JJ, Schambach A, Themis M, Yuan J. Klapwijk JC, et al. Among authors: rothe m. Hum Gene Ther. 2024 Aug;35(15-16):527-542. doi: 10.1089/hum.2024.033. Hum Gene Ther. 2024. PMID: 39049734 Review.
Palbociclib in Patients With Soft Tissue Sarcoma With CDK4 Amplifications: Results From the Targeted Agent and Profiling Utilization Registry Study.
Schuetze S, Rothe M, Mangat PK, Garrett-Mayer E, Meric-Bernstam F, Calfa CJ, Farrington LC, Livingston MB, Wentzel K, Behl D, Kier Y, Marr AS, von Mehren M, Press JZ, Thota R, Grantham GN, Gregory A, Hinshaw DC, Halabi S, Schilsky RL. Schuetze S, et al. Among authors: rothe m. JCO Precis Oncol. 2024 Jul;8:e2400219. doi: 10.1200/PO.24.00219. JCO Precis Oncol. 2024. PMID: 39013131 Clinical Trial.
Multi-omics analysis of diabetic pig lungs reveals molecular derangements underlying pulmonary complications of diabetes mellitus.
Shashikadze B, Flenkenthaler F, Kemter E, Franzmeier S, Stöckl JB, Haid M, Riols F, Rothe M, Pichl L, Renner S, Blutke A, Wolf E, Fröhlich T. Shashikadze B, et al. Among authors: rothe m. Dis Model Mech. 2024 Jul 1;17(7):dmm050650. doi: 10.1242/dmm.050650. Epub 2024 Jul 23. Dis Model Mech. 2024. PMID: 38900131 Free article.
Talazoparib in Patients With Solid Tumors With BRCA1/2 Mutation: Results From the Targeted Agent and Profiling Utilization Registry Study.
Srkalovic G, Rothe M, Mangat PK, Garrett-Mayer E, Ahn ER, Brouse G, Chan J, Mehmi I, Khalil M, Duvivier HL, Gaba A, Leuva H, Thota R, Yost KJ, Grantham GN, Gregory A, Hinshaw DC, Halabi S, Schilsky RL. Srkalovic G, et al. Among authors: rothe m. JCO Precis Oncol. 2024 Jun;8:e2400026. doi: 10.1200/PO.24.00026. JCO Precis Oncol. 2024. PMID: 38865672 Clinical Trial.
438 results