Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

Lindsey A. George, Spencer K. Sullivan, Adam Giermasz, +25

New England Journal of Medicine

CD8+ T-cell responses to adeno-associated virus capsid in humans

Federico Mingozzi, Marcela V. Maus, Daniel J. Hui, +10

Nature Medicine

Cd8+ T-cell ResponsesImmunologyAntiviral ResponseVirologyCd4 T Cell Responses +3

Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

Federico Mingozzi, Xavier M. Anguela, Giulia Pavani, +14

Science Translational Medicine

AAV-1–mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells

Federico Mingozzi, J. J. M. Meulenberg, Daniel J. Hui, +8

Blood

Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

Jeannette Bennicelli, J. Fraser Wright, András M. Komáromy, +17

Molecular Therapy

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Federico Mingozzi, Nicole C. Hasbrouck, Etiena Basner‐Tschakarjan, +9

Blood

Alpha Interferon Induces Distinct Translational Control Programs To Suppress Hepatitis C Virus RNA Replication

Chunfu Wang, Jill Pflugheber, Rhea Sumpter, +4

Journal of Virology

Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors

Gary C. Pien, Etiena Basner‐Tschakarjan, Daniel J. Hui, +8

Journal of Clinical Investigation

Adeno-associated VirusAdeno-associated Viral VectorsViral ReplicationHepatologyViral Hepatitis +12

Distinct Induction Patterns and Functions of Two Closely Related Interferon-inducible Human Genes, ISG54 and ISG56

Fulvia Terenzi, Daniel J. Hui, William C. Merrick, +1

Journal of Biological Chemistry

Viral ReplicationGeneticsImmunologyMolecular BiologyGene Characterization +19

Viral Stress-inducible Protein p56 Inhibits Translation by Blocking the Interaction of eIF3 with the Ternary Complex eIF2·GTP·Met-tRNAi

Daniel J. Hui, C. Ramana Bhasker, William C. Merrick, +1

Journal of Biological Chemistry

Ribosome DissociationViral ReplicationEif3.ternary Complex FormationMolecular VirologyNatural Sciences +11