scholarly journals Adeno-Associated Virus D-Sequence-Mediated Suppression of Expression of a Human Major Histocompatibility Class II Gene: Implications in the Development of Adeno-Associated Virus Vectors for Modulating Humoral Immune Response

2020 ◽  
Vol 31 (9-10) ◽  
pp. 565-574 ◽  
Author(s):  
Hyung-Joo Kwon ◽  
Keyun Qing ◽  
Selvarangan Ponnazhagan ◽  
Xu-Shan Wang ◽  
David M. Markusic ◽  
...  
1989 ◽  
Vol 9 (11) ◽  
pp. 5219-5222
Author(s):  
A Celada ◽  
R Maki

The X box is a loosely conserved DNA sequence that is located upstream of all major histocompatibility class II genes and is one of the cis-acting regulatory elements. Despite the similarity between all X-box sequences, each promoter-proximal X box in the mouse appears to bind a separate nuclear factor.


2018 ◽  
Vol 103 ◽  
pp. 106-114
Author(s):  
Quinn H. Abram ◽  
Kazuhiro Fujiki ◽  
Marije Booman ◽  
Emily Chin-Dixon ◽  
Guang Wei ◽  
...  

2020 ◽  
Vol 21 (10) ◽  
pp. 3433
Author(s):  
Matthew Piechnik ◽  
Kazuki Sawamoto ◽  
Hidenori Ohnishi ◽  
Norio Kawamoto ◽  
Yasuhiko Ago ◽  
...  

The humoral immune response elicited by adeno-associated virus (AAV)-mediated gene therapy for the treatment of mucopolysaccharidoses (MPS) poses a significant challenge to achieving therapeutic levels of transgene expression. Antibodies targeting the AAV capsid as well as the transgene product diminish the production of glycosaminoglycan (GAG)-degrading enzymes essential for the treatment of MPS. Patients who have antibodies against AAV capsid increase in number with age, serotype, and racial background and are excluded from the clinical trials at present. In addition, patients who have undergone AAV gene therapy are often excluded from the additional AAV gene therapy with the same serotype, since their acquired immune response (antibody) against AAV will limit further efficacy of treatment. Several methods are being developed to overcome this immune response, such as novel serotype design, antibody reduction by plasmapheresis and immunosuppression, and antibody evasion using empty capsids and enveloped AAV vectors. In this review, we examine the mechanisms of the anti-AAV humoral immune response and evaluate the strengths and weaknesses of current evasion strategies in order to provide an evidence-based recommendation on evading the immune response for future AAV-mediated gene therapies for MPS.


1996 ◽  
Vol 16 (15) ◽  
pp. 4588-4595 ◽  
Author(s):  
Mehdi Tafti ◽  
Seiji Nishino ◽  
Michael S. Aldrich ◽  
Wennie Liao ◽  
William C. Dement ◽  
...  

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