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Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis.
Caroline J Aalbers, Lisette Bevaart, Scott Loiler et al. · PLoS ONE · 2015
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Solvent Accessibility of Residues Undergoing Pathogenic Variations in Humans: From Protein Structures to Protein Sequences
Castrense Savojardo, Matteo Manfredi, Pier Luigi Martelli et al. · Frontiers in Molecular Biosciences · 2021
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MISTIC: A prediction tool to reveal disease-relevant deleterious missense variants.
Kirsley Chennen, Thomas Weber, Xavière Lornage et al. · PLoS ONE · 2020
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Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy
Jennifer E. Adair, Timothy Waters, Kevin G. Haworth et al. · Nature Communications · 2016
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