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Updated: Aug 18, 2025

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
Published on: August 7, 2015
Vector Shedding and Immunogenicity Sampling in AAV-Based Gene Therapy for Retinitis Pigmentosa Patients
1Institute of Human Nutrition, Columbia University Irving Medical Center, New York, NY, USA. ach4001@med.cornell.edu.
Abstract:
Viral vectors are employed in gene therapy to deliver functional genes. It is often traceable in bodily secretions and excretions of the recipients. Methods of collecting, processing, and storing specimens need to be standardized to ensure effective regulation and monitoring of vector safety. Furthermore, these regulations are crucial in upholding the safety and efficacy of clinical trials and subsequent delivery of these treatments. This chapter discusses vector immunogenicity monitoring and methods of lacrimal, saliva, urine, blood, and serum sampling in patients with retinitis pigmentosa before and throughout the delivery of adeno-associated virus-based gene therapy.

