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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
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
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. Res Sq [Preprint]. 2023 Dec 28:rs.3.rs-3788652. doi: 10.21203/rs.3.rs-3788652/v1. Res Sq. 2023. PMID: 38234818 Free PMC article. Updated. Preprint.
Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2.
Sondhi D, Kaminsky SM, Hackett NR, Pagovich OE, Rosenberg JB, De BP, Chen A, Van de Graaf B, Mezey JG, Mammen GW, Mancenido D, Xu F, Kosofsky B, Yohay K, Worgall S, Kaner RJ, Souwedaine M, Greenwald BM, Kaplitt M, Dyke JP, Ballon DJ, Heier LA, Kiss S, Crystal RG. Sondhi D, et al. Among authors: de bp. Sci Transl Med. 2020 Dec 2;12(572):eabb5413. doi: 10.1126/scitranslmed.abb5413. Sci Transl Med. 2020. PMID: 33268510 Free PMC article. Clinical Trial.
84 results