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INCEPTUS Natural History, Run-in Study for Gene Replacement Clinical Trial in X-Linked Myotubular Myopathy.
Dowling JJ, Müller-Felber W, Smith BK, Bönnemann CG, Kuntz NL, Muntoni F, Servais L, Alfano LN, Beggs AH, Bilder DA, Blaschek A, Duong T, Graham RJ, Jain M, Lawlor MW, Lee J, Coats J, Lilien C, Lowes LP, MacBean V, Neuhaus S, Noursalehi M, Pitts T, Finlay C, Christensen S, Rafferty G, Seferian AM, Tsuchiya E, James ES, Miller W, Sepulveda B, Vila MC, Prasad S, Rico S, Shieh PB; INCEPTUS investigators. Dowling JJ, et al. Among authors: lee j. J Neuromuscul Dis. 2022;9(4):503-516. doi: 10.3233/JND-210781. J Neuromuscul Dis. 2022. PMID: 35694931 Free PMC article.
Safety and efficacy of gene replacement therapy for X-linked myotubular myopathy (ASPIRO): a multinational, open-label, dose-escalation trial.
Shieh PB, Kuntz NL, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Servais L, Smith BK, Muntoni F, Blaschek A, Foley AR, Saade DN, Neuhaus S, Alfano LN, Beggs AH, Buj-Bello A, Childers MK, Duong T, Graham RJ, Jain M, Coats J, MacBean V, James ES, Lee J, Mavilio F, Miller W, Varfaj F, Murtagh M, Han C, Noursalehi M, Lawlor MW, Prasad S, Rico S. Shieh PB, et al. Among authors: lee j. Lancet Neurol. 2023 Dec;22(12):1125-1139. doi: 10.1016/S1474-4422(23)00313-7. Lancet Neurol. 2023. PMID: 37977713 Free article. Clinical Trial.
Effects of gene replacement therapy with resamirigene bilparvovec (AT132) on skeletal muscle pathology in X-linked myotubular myopathy: results from a substudy of the ASPIRO open-label clinical trial.
Lawlor MW, Schoser B, Margeta M, Sewry CA, Jones KA, Shieh PB, Kuntz NL, Smith BK, Dowling JJ, Müller-Felber W, Bönnemann CG, Seferian AM, Blaschek A, Neuhaus S, Foley AR, Saade DN, Tsuchiya E, Qasim UR, Beatka M, Prom MJ, Ott E, Danielson S, Krakau P, Kumar SN, Meng H, Vanden Avond M, Wells C, Gordish-Dressman H, Beggs AH, Christensen S, Conner E, James ES, Lee J, Sadhu C, Miller W, Sepulveda B, Varfaj F, Prasad S, Rico S. Lawlor MW, et al. Among authors: lee j. EBioMedicine. 2024 Jan;99:104894. doi: 10.1016/j.ebiom.2023.104894. Epub 2023 Dec 12. EBioMedicine. 2024. PMID: 38086156 Free PMC article.
A proof-of-concept study for precise mapping of pigmented basal cell carcinoma in asian skin using multispectral optoacoustic tomography imaging with level set segmentation.
Li X, Teo VX, Kwa CY, Ebrahim Attia AB, Bi R, Chuah SY, Tan MWP, Chia HY, Chua SH, Lee JXS, Cheng SWN, Dinish US, Thng STG, Olivo M. Li X, et al. Among authors: lee jxs. Eur J Nucl Med Mol Imaging. 2025 Jan 24. doi: 10.1007/s00259-025-07072-x. Online ahead of print. Eur J Nucl Med Mol Imaging. 2025. PMID: 39849150
Paediatric-specific content in data standards for health.
Turner MA, Kalra D, Cornet R, Palmeri A, Sen A, Owen J, Pansieri C, Lee J, Hedley V, Nally S, Bonifazi F, Leary R, Straub V. Turner MA, et al. Among authors: lee j. Arch Dis Child. 2025 Jan 23:archdischild-2024-327931. doi: 10.1136/archdischild-2024-327931. Online ahead of print. Arch Dis Child. 2025. PMID: 39848700 No abstract available.
Interferon regulatory factor 2 of red-spotted grouper (Epinephelus akaara): insights into its transcriptional profiling, antiviral potential, and function in macrophage polarization.
Sirisena DMKP, Kim G, Warnakula WADLR, Jayamali BPMV, Tharanga EMT, Jayasinghe JDHE, Sandeepani RI, Wan Q, Sohn H, Lee J. Sirisena DMKP, et al. Among authors: lee j. Dev Comp Immunol. 2025 Jan 21:105323. doi: 10.1016/j.dci.2025.105323. Online ahead of print. Dev Comp Immunol. 2025. PMID: 39848353
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