Enhancement of liver-directed transgene expression at initial and repeat doses of AAV vectors admixed with ImmTOR nanoparticles

Gene Therapy

(2019) Katherine A. High et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation–Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials

(2019) Albert M. Maguire et al.   OPHTHALMOLOGY

Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?

(2019) Allison M. Keeler et al.   Annual Review of Virology

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor

(2018) Amanda M. Dudek et al.   JOURNAL OF VIROLOGY

Tolerogenic nanoparticles restore the antitumor activity of recombinant immunotoxins by mitigating immunogenicity

(2018) Ronit Mazor et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

(2018) Pasqualina Colella et al.   Molecular Therapy-Methods & Clinical Development

An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

(2018) Junjiang Sun et al.   Molecular Therapy-Methods & Clinical Development

Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration

(2018) Amine Meliani et al.   Nature Communications

Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors

(2017) Marianna Hösel et al.   HEPATOLOGY

Gene Therapy for Hemophilia

(2017) Arthur W. Nienhuis et al.   MOLECULAR THERAPY

Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy

(2017) Jerry R. Mendell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tolerogenic nanoparticles to induce immunologic tolerance: Prevention and reversal of FVIII inhibitor formation

(2016) Ai-Hong Zhang et al.   CELLULAR IMMUNOLOGY

An essential receptor for adeno-associated virus infection

(2016) S. Pillay et al.   NATURE

Improving the efficacy and safety of biologic drugs with tolerogenic nanoparticles

(2016) Takashi K. Kishimoto et al.   Nature Nanotechnology

Strategies to circumvent humoral immunity to adeno-associated viral vectors

(2015) Longping V Tse et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy

(2015) Randy J. Chandler et al.   JOURNAL OF CLINICAL INVESTIGATION

mTORC1-Induced HK1-Dependent Glycolysis Regulates NLRP3 Inflammasome Activation

(2015) Jong-Seok Moon et al.   Cell Reports

In Silico Reconstruction of the Viral Evolutionary Lineage Yields a Potent Gene Therapy Vector

(2015) Eric Zinn et al.   Cell Reports

Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

(2015) Manuela Corti et al.   Human Gene Therapy Clinical Development

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Polymeric synthetic nanoparticles for the induction of antigen-specific immunological tolerance

(2014) Roberto A. Maldonado et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

(2014) M Corti et al.   Molecular Therapy-Methods & Clinical Development

Immune responses to AAV vectors: overcoming barriers to successful gene therapy

(2013) F. Mingozzi et al.   BLOOD

Immune Responses to AAV in Clinical Trials

(2011) Federico Mingozzi et al.   CURRENT GENE THERAPY

Autophagy Controls IL-1β Secretion by Targeting Pro-IL-1β for Degradation

(2011) James Harris et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

(2011) Virginie Monteilhet et al.   MOLECULAR THERAPY

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

BALB/c Mice Show Impaired Hepatic Tolerogenic Response Following AAV Gene Transfer to the Liver

(2010) Ekaterina Breous et al.   MOLECULAR THERAPY

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

(2009) F. Mingozzi et al.   BLOOD

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

(2009) Gary C. Pien et al.   JOURNAL OF CLINICAL INVESTIGATION

Gene Delivery to the Juvenile Mouse Liver Using AAV2/8 Vectors

(2008) Sharon C Cunningham et al.   MOLECULAR THERAPY