Validation of an IFN-gamma ELISpot assay to measure cellular immune responses against viral antigens in non-human primates

AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer

(2020) Helena Costa Verdera et al.   MOLECULAR THERAPY

Tetramer-Based Enrichment of Preexisting Anti-AAV8 CD8+ T Cells in Human Donors Allows the Detection of a TEMRA Subpopulation

(2020) Céline Vandamme et al.   Frontiers in Immunology

State-of-the-Art 2019 on Gene therapy for Phenylketonuria

(2019) Hiu Man Grisch-Chan et al.   HUMAN GENE THERAPY

The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy

(2019) Brian R. Long et al.   Molecular Therapy-Methods & Clinical Development

Prevalence of Anti-Adeno-Associated Virus Immune Responses in International Cohorts of Healthy Donors

(2019) Anita Kruzik et al.   Molecular Therapy-Methods & Clinical Development

2019 White Paper on Recent Issues in Bioanalysis: FDA Immunogenicity Guidance, Gene Therapy, Critical Reagents, Biomarkers and Flow Cytometry Validation (Part 3 – Recommendations on 2019 FDA Immunogenicity Guidance, Gene Therapy Bioanalytical Challenges, Strategies for Critical Reagent Management, Biomarker Assay Validation, Flow Cytometry Validation & CLSI H62)

(2019) Steven Piccoli et al.   Bioanalysis

Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model

(2018) Kasun Kodippili et al.   HUMAN GENE THERAPY

Assessment of Humoral, Innate, and T-Cell Immune Responses to Adeno-Associated Virus Vectors

(2018) Roberto Calcedo et al.   Human Gene Therapy Methods

Emerging Issues in AAV-Mediated In Vivo Gene Therapy

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

AAV Immunogenicity: A Matter of Sensitivity

(2018) Federico Mingozzi   MOLECULAR THERAPY

Exposure to wild-type AAV drives distinct capsid immunity profiles in humans

(2018) Klaudia Kuranda et al.   JOURNAL OF CLINICAL INVESTIGATION

Strategy to detect pre-existing immunity to AAV gene therapy

(2017) L Falese et al.   GENE THERAPY

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

(2017) Lindsey A. George et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape

(2017) Federico Mingozzi et al.   Annual Review of Virology

Research Techniques Made Simple: Monitoring of T-Cell Subsets using the ELISPOT Assay

(2016) Christian Möbs et al.   JOURNAL OF INVESTIGATIVE DERMATOLOGY

Validation of an IFNγ/IL2 FluoroSpot assay for clinical trial monitoring

(2016) Nina Körber et al.   Journal of Translational Medicine

2016 White Paper on recent issues in bioanalysis: focus on biomarker assay validation (BAV): (Part 3 – LBA, biomarkers and immunogenicity)

(2016) Susan Richards et al.   Bioanalysis

Enhanced immunogenicity of a respiratory syncytial virus (RSV) F subunit vaccine formulated with the adjuvant GLA-SE in cynomolgus macaques

(2015) Kathryn Patton et al.   VACCINE

A White Paper—Consensus and Recommendations of a Global Harmonization Team on Assessing the Impact of Immunogenicity on Pharmacokinetic Measurements

(2014) J. M. Sailstad et al.   AAPS Journal

Enzyme-Linked Immunospot Assay for Detection of Human Respiratory Syncytial Virus F Protein-Specific Gamma Interferon-Producing T Cells

(2014) Kathryn Patton et al.   Clinical and Vaccine Immunology

Our Journey to Successful Gene Therapy for Hemophilia B

(2014) Amit C. Nathwani et al.   HUMAN GENE THERAPY

T cell responses: naïve to memory and everything in between

(2013) Nathan D. Pennock et al.   ADVANCES IN PHYSIOLOGY EDUCATION

Anti-Dystrophin T Cell Responses in Duchenne Muscular Dystrophy: Prevalence and a Glucocorticoid Treatment Effect

(2013) Kevin M. Flanigan et al.   HUMAN GENE THERAPY

ELISpot for measuring human immune responses to vaccines

(2011) Meredith Slota et al.   Expert Review of Vaccines

Recommendations for the validation of cell-based assays used for the detection of neutralizing antibody immune responses elicited against biological therapeutics

(2011) Shalini Gupta et al.   JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS

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

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

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

Immune Responses to AAV in Canine Muscle Monitored by Cellular Assays and Noninvasive Imaging

(2009) Zejing Wang et al.   MOLECULAR THERAPY

Precision and linearity targets for validation of an IFNγ ELISPOT, cytokine flow cytometry, and tetramer assay using CMV peptides

(2008) Holden T Maecker et al.   BMC IMMUNOLOGY

Recommendations for the validation of immunoassays used for detection of host antibodies against biotechnology products

(2008) Gopi Shankar et al.   JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS