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Gene therapy

Low efficacy of recombinant SV40 in Ugt1a1^-/- mice with severe inherited hyperbilirubinemia

Xiaoxia Shi, Giulia Bortolussi, [ ... ], Piter J. Bosma

Supervised machine learning for automated classification of human Wharton’s Jelly cells and mechanosensory hair cells

Abihith Kothapalli, Hinrich Staecker, Adam J. Mellott

“I would really want to know that they had my back”: Transgender women’s perceptions of HIV cure-related research in the United States

Tonia Poteat, Anushka Aqil, [ ... ], Karine Dubé

The protective mutation A673T in amyloid precursor protein gene decreases Aβ peptides production for 14 forms of Familial Alzheimer’s Disease in SH-SY5Y cells

Antoine Guyon, Joël Rousseau, Gabriel Lamothe, Jacques P. Tremblay

Inhibitors of metalloprotease, γ-sectretase, protein kinase C and Rho kinase inhibit wild-type adenoviral replication

Alice Liu, Cristhian J. Ildefonso, [ ... ], Richard L. Hurwitz

FDA orphan drug designations for lysosomal storage disorders – a cross-sectional analysis

Sven F. Garbade, Matthias Zielonka, [ ... ], Markus Ries

Adeno-associated virus-mediated expression of human butyrylcholinesterase to treat organophosphate poisoning

Vibhor Gupta, C. Linn Cadieux, [ ... ], James M. Wilson

Evaluation of suitable reference genes for gene expression analysis in the northern root-knot nematode, Meloidogyne hapla

Xiaojing Wu, Hongyan Yu, [ ... ], Yuxi Duan

Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents

Holly Landrum Peay, Ryan Fischer, [ ... ], Carol Mansfield

Evaluation of the awareness of novel advanced therapies among family medicine residents in Spain

Miguel Sola, Carmen Sanchez-Quevedo, [ ... ], Fernando Campos

Pre-existing antibodies to candidate gene therapy vectors (adeno-associated vector serotypes) in domestic cats

Pengfei Li, Eva Boenzli, Regina Hofmann-Lehmann, A. Katrin Helfer-Hungerbuehler

mRNA transfection by a Xentry-protamine cell-penetrating peptide is enhanced by TLR antagonist E6446

Glenn D. Bell, Yi Yang, Euphemia Leung, Geoffrey W. Krissansen

Development and testing of AAV-delivered single-chain variable fragments for the treatment of methamphetamine abuse

Charles E. Hay, Guillermo A. Gonzalez III, [ ... ], Eric C. Peterson

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