Immunoadsorption Plasmapheresis for the Removal of Plasma Immunoglobulins to Enable Repeat Dose Administration with an AAV5 Gene Therapy Vector

被引：0

作者：

Long, Brian R. ^{[1
]}

Hock, Benjamin M. ^{[1
]}

O'Neill, Charles A. ^{[1
]}

Arens, Jeremy ^{[1
]}

Seitel, Theresa ^{[1
]}

Crockett, Lucy ^{[1
]}

Relouzat, Francis ^{[2
]}

Fovet, Claire-Maelle ^{[2
]}

Dereuddre-Bosquet, Nathalie ^{[2
]}

Maisonnasse, Pauline ^{[2
]}

Letscher, Helene ^{[2
]}

Le Grand, Roger ^{[2
]}

Vettermann, Christian ^{[1
]}

Gupta, Soumi ^{[1
]}

机构：

[1] BioMarin Pharmaceut Inc, Novato, CA USA

[2] Inst Biol Francois Jacob, IMVA IDMIT Ctr DRF, Commissariat Energie Atom & Energies Alternat, Fontenay Aux Roses, France

来源：

MOLECULAR THERAPY | 2023年 / 31卷 / 04期

关键词：

D O I：

暂无

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

1551

引用

页码：729 / 730

页数：2

共 6 条

[1] Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye
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[2] Successful Repeated Hepatic Gene Delivery in Mice and Non-Human Primates Achieved by Sequential Administration of AAV5 and AAV1 Vector Serotypes
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[3] Evaluation of prophylactic immune suppression and application to repeat dose administration of AAV-mediated gene therapy in mice
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[4] Activity of transgene-produced B-domain-deleted factor VIII in human plasma following AAV5 gene therapy
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Srimani, Jaydeep
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[5] FIRST RESULTS FROM A DOSE-ESCALATING STUDY WITH AAV5 VECTOR CONTAINING WILD TYPE HUMAN FACTOR IX GENE THERAPY IN PATIENTS WITH SEVERE OR MODERATELY-SEVERE HAEMOPHILIA B
Leebeek, F.
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Meijer, K.
Coppens, M.
Kampmann, P.
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HAEMATOLOGICA, 2016, 101 : 169 - 169
[6] Interim analysis of immunogenicity to the vector capsid and transgene-expressed human FVIII IN a phase-1/2 clinical study of BMN-270, an AAV5 mediated gene therapy for hemophilia A
Long, B.
Kim, B.
Wong, W. Y.
Yang, K.
Vettermann, C.
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Hardet, R.
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