Paper Review Records
All Paper Review Records
10 valid samples · Newest publication first
Review days = acceptance date − received date. PDF, DOI, and publisher-page sources are retained.
Inhibition of immune response reduces pathology in dorsal root ganglia and peripheral nerves in cynomolgus macaques following AAV gene therapy
AuthorsBranka Grubor; Kate L. Henry; Su Jing Chan; Mark Sheehan; Anumeha Shah; Alex Pellerin; Judy Bai; Prasad Nadella; Santhosh Bommegowda; Patrick Cullen; Eric Tien; Vivian Chen; Nicholas P. van der Munnik; Stephanie White-Hunt; Edward D. Plowey; Stefan Hamann; Amanda J. Guise; Shanqin Xu; Melissa Kirkland; Jessica Doherty; Eugenia Lyashenko; Guruharsha Bhat; Kelly Glajch; Shih-Ching Lo; Davide Gianni; Pete Clarner; Jake Gagnon; Jenhwa Chu; Kalyani Nambiar; Mukesh Lulla; Fengmei Zheng; Asmerom Weldeab; Dan Bartlett; Amos Gutnick; Taylor Reynolds; Kan Zhu; Dann Huh; Thomas M. Carlile; James Fikes; Patrick Trapa; Junghae Suh; Dale Morris; Linda Burkly
AffiliationsBiogen, Cambridge, MA 02142, USA; Charles River Laboratories, Durham, NC 27703, USA
Leveraging the active conformation of LFA-1 as a potential target for hematological malignancies
AuthorsAbhishek Puthenveetil; Guy Biber; Batel Sabag; Meirav Kedmi; Avigdor Abraham; Noah Joseph; Mira Barda-Saad
AffiliationsThe Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat-Gan 5290002, Israel; The Chaim Sheba Medical Center, Tel-Hashomer, Ramat-Gan, Israel; Sackler School of Medicine, Tel- Aviv University, Tel-Aviv, Israel
AAV-PHP.eB achieves superior neuronal transduction over AAV9 in pigtail macaques following intracerebroventricular administration
AuthorsMichal G. Fortuna; Lyle H. Nyberg; Naz Taskin; Avery Hunker; Natalie Weed; Kathryn Gudsnuk; Melissa Berg; Boaz P. Levi; Ed Lein; Jonathan T. Ting
AffiliationsAllen Institute for Brain Science, Seattle, WA, USA; Washington National Primate Research Center, Seattle, WA, USA; Department of Neurological Surgery, University of Washington, Seattle, WA, USA; Department of Neurobiology & Biophysics, University of Washington, Seattle, WA, USA
Nonclinical toxicity study duration in AAV gene therapy development: Evidence from industry survey supports adequacy of short-term assessments
AuthorsBasel T. Assaf; David Edwards; Shawn Jay Berens; Brian Lauritzen; Christelle Pierrot; Francis Fonyuy Tukov; Cristina Bertinetti-Lapatki; Manuela Braun; Kai Schaefer; Jessica L. Lynch
AffiliationsSanofi, Cambridge, MA 02141, USA; UCB Pharma, Slough, UK; Eli Lilly and Company, Indianapolis, IN 46285, USA; Novo Nordisk A/S, Maaloev, Denmark; Astellas Pharma B.V., Leiden, the Netherlands; Novartis Pharmaceuticals Corporation, East Hanover, NJ 07936, USA; Roche Innovation Center, 4070 Basel, Switzerland; Bayer AG, 13342 Berlin, Germany; AbbVie Deutschland GmbH & Co. KG, Ludwigshafen, Germany; Johnson & Johnson Innovative Medicine, Spring House, PA 19477, USA
Gene therapy supports long-term reconstitution of patient hematopoietic stem cells in deficiency of adenosine deaminase 2
AuthorsChiara Rigamonti; Dimitri Bulté; Federica Barzaghi; Cristina Mesa-Nuñez; Luca Basso-Ricci; Emanuela Pettinato; Camilla Visconti; Sara Degl’Innocenti; Francesco Gazzo; Fabrizio Benedicenti; Sergio Arévalo; Pamela Quaranta; Francesca Sanvito; Roberta Caorsi; Francesca Schena; Michela Lupia; Silvia Federici; Antonella Insalaco; Fabrizio De Benedetti; Serena Scala; Maurizio Miano; Francesca Conti; Maria Pia Cicalese; Eugenio Montini; Marco Gattorno; Carlo Dufour; Alessandro Aiuti; Alessandra Mortellaro
AffiliationsSan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy; Vita-Salute San Raffaele University, 20132 Milan, Italy; Pediatric Immunohematology and Bone Marrow Transplantation Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy; GLP Test Facility, San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy; Pathology Unit, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy; Department of Electronics, Information and Bioengineering, Politecnico di Milano, 20133 Milan, Italy; UOC Rheumatology and Autoinflammatory Diseases, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy; Hematology Unit, IRCCS Istituto Giannina Gaslini, 16147 Genoa, Italy; Division of Rheumatology, Bambino Gesù Children’s Hospital, ERN-RITA Center, IRCCS, 00165 Rome, Italy; Pediatric Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, 40138 Bologna, Italy; Department of Medical and Surgical Sciences, Alma Mater Studiorum, University of Bologna, 40126 Bologna, Italy
SLC20A1 knockout to stably express a fusogenic gibbon ape leukemia virus envelope glycoprotein for lentiviral vector production
AuthorsRodrigo José Nogueira; Ana Filipa Rodrigues; Daniel Alexandre Mestre; Ana Sofia Coroadinha
AffiliationsiBET - Instituto de Biologia Experimental e Tecnológica, Apartado 12, 2781-901 Oeiras, Portugal; Instituto de Tecnologia Química e Biológica António Xavier, Universidade Nova de Lisboa, Av. da República, 2780-157 Oeiras, Portugal; LASIGE, Faculdade de Ciências da Universidade de Lisboa, 1749-016 Lisboa, Portugal
Transcriptional changes in non-human primate tissues after intrathecal delivery of serotype 9 adeno-associated viral vector: Insights into organ toxicities
AuthorsFumiaki Aihara; Matthew Mardo; Vera Ruda; Tony del Rio; Karine Bigot; Jochen Singer; Megumi Onishi-Seebacher; Philippe Couttet; Keith Mansfield; Eloise Hudry
AffiliationsNovartis Biomedical Research, Cambridge, MA, USA; Novartis Biomedical Research, Basel, Switzerland; Novartis Biomedical Research, San Diego, CA, USA
Therapeutic mRNA delivery of CRISPR-Cas9 to the trabecular meshwork reverses ocular hypertension in myocilin glaucoma
AuthorsSam Yacoub; Balasankara Reddy Kaipa; Linya Li; Sarahi Rios; Ramesh Kasetti; Prabhavathi Maddineni; Abbot F. Clark; Gulab S. Zode
AffiliationsDepartment of Pharmacology and Neuroscience and North Texas Eye Research Institute, University of North Texas Health Science Center, Fort Worth, TX 76107, USA; Gavin Herbert Eye Institute-Center for Translational Vision Research, Department of Ophthalmology, Department of Physiology and Biophysics, University of California, Irvine, Irvine School of Medicine, Irvine, CA 92697, USA; Department of Ophthalmology, School of Medicine, University of Missouri, Columbia, MO 65212, USA
Bedside manufacturing of engineered stem cells using gene therapy foam
AuthorsCarrie L. Cummings; Sirkka B. Stephan; Katelyn Fitzgerald; Matthias T. Stephan
AffiliationsTranslational Science and Therapeutics Division, Fred Hutchinson Cancer Center, Seattle, WA 98109, USA; Department of Bioengineering and Molecular Engineering & Sciences Institute, University of Washington, Seattle, WA 98109, USA; Department of Medicine, Division of Medical Oncology, University of Washington, Seattle, WA 98195, USA
A potency and toxicology study on tolerability of cryopreserved mesenchymal stem cell product with DMSO in septic mice and nude rats
AuthorsYan Wang; Glinton Hanover; Aidan B.P. Murray; Mahmoud Salkhordeh; Yuan Tan; Chi Wang; Jia-Pey Wang; Andrea McRae; Yupu Deng; Maria Florian; Pramod Sahadevan; Duncan J. Stewart; Lauralyn Mclntyre; Luciana Souza-Moreira; Shirley H.J. Mei
AffiliationsRegenerative Medicine Program, Ottawa Hospital Research Institute, Ottawa, ON, Canada; Clinical Epidemiology Program, Ottawa Hospital Research Institute, Ottawa, ON, Canada; Department of Medicine, University of Ottawa, Ottawa, ON, Canada
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