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Page 1
Expression and processing of mature human frataxin after gene therapy in mice.
Rojsajjakul T, Selvan N, De B, Rosenberg JB, Kaminsky SM, Sondhi D, Janki P, Crystal RG, Mesaros C, Khanna R, Blair IA. Rojsajjakul T, et al. Among authors: de b. Res Sq [Preprint]. 2023 Dec 28:rs.3.rs-3788652. doi: 10.21203/rs.3.rs-3788652/v1. Res Sq. 2023. Update in: Sci Rep. 2024 Apr 10;14(1):8391. doi: 10.1038/s41598-024-59060-0 PMID: 38234818 Free PMC article. Updated. Preprint.
Safety of Intravenous Administration of an AAV8 Vector Coding for an Oxidation-Resistant Human α1-Antitrypsin for the Treatment of α1-Antitrypsin Deficiency.
Rosenberg JB, De BP, Greco A, Gorman N, Kooner V, Chen A, Yost-Bido M, Munoz-Zuluaga C, Kaminsky SM, Rostami M, Monette S, Crystal RG, Sondhi D. Rosenberg JB, et al. Among authors: de bp. Hum Gene Ther. 2023 Feb;34(3-4):139-149. doi: 10.1089/hum.2022.192. Hum Gene Ther. 2023. PMID: 36606685 Free PMC article.
Identification of Safe and Effective Intravenous Dose of AAVrh.10hFXN to Treat the Cardiac Manifestations of Friedreich's Ataxia.
Munoz-Zuluaga C, Gertz M, Yost-Bido M, Greco A, Gorman N, Chen A, Kooner V, Rosenberg JB, De BP, Kaminsky SM, Borczuk A, Ricart Arbona RJ, Martin HR, Monette S, Khanna R, Barth JA, Crystal RG, Sondhi D. Munoz-Zuluaga C, et al. Among authors: de bp. Hum Gene Ther. 2023 Jul;34(13-14):605-615. doi: 10.1089/hum.2023.020. Epub 2023 Jul 4. Hum Gene Ther. 2023. PMID: 37166361 Free PMC article.
Positron Emission Tomography Quantitative Assessment of Off-Target Whole-Body Biodistribution of I-124-Labeled Adeno-Associated Virus Capsids Administered to Cerebral Spinal Fluid.
Rosenberg JB, Fung EK, Dyke JP, De BP, Lou H, Kelly JM, Reejhsinghani L, Ricart Arbona RJ, Sondhi D, Kaminsky SM, Cartier N, Hinderer C, Hordeaux J, Wilson JM, Ballon DJ, Crystal RG. Rosenberg JB, et al. Among authors: de bp. Hum Gene Ther. 2023 Nov;34(21-22):1095-1106. doi: 10.1089/hum.2023.060. Epub 2023 Oct 23. Hum Gene Ther. 2023. PMID: 37624734 Free PMC article.
Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease.
De BP, Rosenberg JB, Selvan N, Wilson I, Yusufzai N, Greco A, Kaminsky SM, Heier LA, Ricart Arbona RJ, Miranda IC, Monette S, Nair A, Khanna R, Crystal RG, Sondhi D. De BP, et al. Hum Gene Ther. 2023 Sep;34(17-18):905-916. doi: 10.1089/hum.2023.067. Hum Gene Ther. 2023. PMID: 37624739 Free PMC article.
Performance Comparison of 10 State-of-the-Art Machine Learning Algorithms for Outcome Prediction Modeling of Radiation-Induced Toxicity.
Salazar RM, Nair SS, Leone AO, Xu T, Mumme RP, Duryea JD, De B, Corrigan KL, Rooney MK, Ning MS, Das P, Holliday EB, Liao Z, Court LE, Niedzielski JS. Salazar RM, et al. Among authors: de b. Adv Radiat Oncol. 2024 Nov 13;10(2):101675. doi: 10.1016/j.adro.2024.101675. eCollection 2025 Feb. Adv Radiat Oncol. 2024. PMID: 39717195 Free PMC article.
459 results