Work

Harnessing B19-directed CAR T cells for AAV vector administration in seropositive patients: The importance of the niche

Year: 2025

Type: article

Abstract: Adeno-associated virus (AAV) vectors have significantly advanced in vivo gene therapy for monogenic diseases. Humoral immunity to AAVs represents a major challenge toward the application of this gene transfer modality in the larger population. Pre-existing neutralizing antibodies (Nabs) to AAV capsids reduce the number of patients eligible for treatment, while post-treatment humoral immunity prevents vector re-administration. Addressing this challenge remains a critical need in the field. In this issue of Molecular Therapy, Doshi et al.

Source: Molecular Therapy

DOI: 10.1016/j.ymthe.2025.06.007

Authors: Hanadi Saliba, David Alexandre Gross, Giuseppe Ronzitti

Institutions: Inserm, Université Paris-Saclay, Genethon (France), Université d'Évry Val-d'Essonne

Language: English

Topic: Virus-based gene therapy research

Subfield: Genetics

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $4,500