AAV Gene transfer - Europe PMC

Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients.

One of the main objectives of in vivo gene transfer is to produce therapeutic transgenes in terminally differentiated cells. Gene transfer in multiple tissues is among the best options for gene transfer in multiple organs, according to the extensive medical research and the recent approval of Luxturna ® and Zolgensma ® place vectors derived from adeno-associated viruses. A large number of AAV infections in the human race has emerged as a result because of the virus's wild-type AAV infection, anti-AAV antibodies have evolved and may neutralize the vectors that have been derived from the virus. AAV injection in humans is generally well tolerated, but after the recognition of AAV vector capsid and genome, the immune system can be activated. After the first injection, the production of high-titer neutralizing antibodies to AAV prevents vector re-administration.

Source link: https://europepmc.org/article/MED/35464422

Therapeutic Applications of Adeno-Associated Virus (AAV) Gene Transfer of HLA-G in the Eye.

The aim of this paper is to investigate human leukocyte antigen G in the eye, its role in immune tolerance, and the potential therapeutic use of AAV gene transfer and expression of HLA-G in various ocular tissues.

Source link: https://europepmc.org/article/MED/35408825

Testing preexisting antibodies prior to AAV gene transfer therapy: rationale, lessons and future considerations.

Given the increasing number of gene transfer therapy studies, whether completed or ongoing, preexisting adaptive immunity to the viral vectors used is becoming more important. Antibodies created against viral vectors obtained in a previous exposure to wild-type virus can also harm transgene expression by blocking transduction, limiting the therapeutic effectiveness of the gene transfer therapy but also raising safety concerns. Binding assays that focus on total antibodies and neutralizing antibodies are used in two separate assays: binding assays that measure neutralizing antibodies and neutralizing antibodies.

Source link: https://europepmc.org/article/MED/35356756

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