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Introduction to <fc>AAV</fc> ‐based <i>in vivo</i> Gene Therapy

Recent Development in <i>in vivo</i> Clinical Gene Therapy Platforms

Biomarker and Bioanalytical Readouts for the Development of <fc>AAV</fc> Gene Therapy

Nonclinical and Clinical Study Considerations for Biodistribution, Shedding, and Pharmacokinetics/Pharmacodynamics

Immunogenicity of <fc>AAV</fc> Gene Therapy Products

Bioanalytical Methods to Detect Preexisting and Post‐administration Humoral Immune Responses Against <fc>AAV</fc> Capsid Proteins

Bioanalytical Methods to Study Biodistribution and Shedding of <fc>AAV</fc> ‐Based Gene Therapy Vectors

Transgene <fc>mRNA</fc> Expression Analysis

Quantification of Transgene Protein Expression and Biochemical Function

Substrate and Distal Pharmacodynamic Biomarker Measurements for Gene Therapy

Detection of Cellular Immunity to Viral Capsids and Transgene Proteins

Detection of Humoral Response to Transgene Protein and Gene Editing Reagents

rAAV Integration: Detection and Risk Assessment

Detection and Quantification of Genome Editing Events in Preclinical and Clinical Studies

Introduction to Companion Diagnostics for Gene Therapy

Validation for Gene Therapy Companion Diagnostics

Regulatory Considerations for Gene Therapy Companion Diagnostics

Current Regulatory Landscape for Gene Therapy Product Development and the Role of Biomarkers

Index

Front Matter