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Phenotypic Correction of Murine Mucopolysaccharidosis Type II by Engraftment of Ex Vivo Lentiviral Vector-Transduced Hematopoietic Stem and Progenitor Cells.
Smith MC, Belur LR, Karlen AD, Erlanson O, Podetz-Pedersen KM, McKenzie J, Detellis J, Gagnidze K, Parsons G, Robinson N, Labarre S, Shah S, Furcich J, Lund TC, Tsai HC, McIvor RS, Bonner M. Smith MC, et al. Among authors: karlen ad. Hum Gene Ther. 2022 Dec;33(23-24):1279-1292. doi: 10.1089/hum.2022.141. Epub 2022 Nov 23. Hum Gene Ther. 2022. PMID: 36226412 Free PMC article.
Lentivirus Mediated Correction of Artemis-Deficient Severe Combined Immunodeficiency.
Punwani D, Kawahara M, Yu J, Sanford U, Roy S, Patel K, Carbonaro DA, Karlen AD, Khan S, Cornetta K, Rothe M, Schambach A, Kohn DB, Malech HL, McIvor RS, Puck JM, Cowan MJ. Punwani D, et al. Among authors: karlen ad. Hum Gene Ther. 2017 Jan;28(1):112-124. doi: 10.1089/hum.2016.064. Epub 2016 Sep 7. Hum Gene Ther. 2017. PMID: 27611239 Free PMC article.
AAV-mediated gene transfer to colon-innervating primary afferent neurons.
Gore R, Esmail T, Pflepsen K, Marron Fernandez de Velasco E, Kitto KF, Riedl MS, Karlen A, McIvor RS, Honda CN, Fairbanks CA, Vulchanova L. Gore R, et al. Front Pain Res (Lausanne). 2023 Aug 4;4:1225246. doi: 10.3389/fpain.2023.1225246. eCollection 2023. Front Pain Res (Lausanne). 2023. PMID: 37599864 Free PMC article.
Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice.
Smith MC, Belur LR, Karlen AD, Erlanson O, Furcich J, Lund TC, Seelig D, Kitto KF, Fairbanks CA, Kim KH, Buss N, McIvor RS. Smith MC, et al. Among authors: karlen ad. Mol Ther Methods Clin Dev. 2024 Jan 30;32(1):101201. doi: 10.1016/j.omtm.2024.101201. eCollection 2024 Mar 14. Mol Ther Methods Clin Dev. 2024. PMID: 38374962 Free PMC article.