scholarly journals OneBac: Platform for Scalable and High-Titer Production of Adeno-Associated Virus Serotype 1–12 Vectors for Gene Therapy

2014 ◽  
Vol 25 (3) ◽  
pp. 212-222 ◽  
Author(s):  
Mario Mietzsch ◽  
Sabrina Grasse ◽  
Catherine Zurawski ◽  
Stefan Weger ◽  
Antonette Bennett ◽  
...  
Keyword(s):  
Gene Therapy ◽  
High Titer ◽  
Adeno Associated Virus ◽  
Virus Serotype ◽  
Serotype 1

scholarly journals A phase I trial of adeno-associated virus serotype 1-γ-sarcoglycan gene therapy for limb girdle muscular dystrophy type 2C

Brain ◽  
2011 ◽  
Vol 135 (2) ◽  
pp. 483-492 ◽  
Author(s):  
Serge Herson ◽  
Faycal Hentati ◽  
Aude Rigolet ◽  
Anthony Behin ◽  
Norma B. Romero ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Muscular Dystrophy ◽  
Phase I ◽  
Phase I Trial ◽  
Limb Girdle Muscular Dystrophy ◽  
Adeno Associated Virus ◽  
Virus Serotype ◽  
Serotype 1

scholarly journals Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations

2020 ◽  
Vol 7 (10) ◽  
pp. 1843-1853 ◽  
Author(s):  
Christian Hinderer ◽  
Rod Miller ◽  
Cecilia Dyer ◽  
Julia Johansson ◽  
Peter Bell ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Adeno Associated Virus ◽  
Virus Serotype ◽  
Serotype 1

scholarly journals Complete Correction of Brain and Spinal Cord Pathology in Metachromatic Leukodystrophy Mice

2021 ◽  
Vol 14 ◽  
Author(s):  
Emilie Audouard ◽  
Valentin Oger ◽  
Béatrix Meha ◽  
Nathalie Cartier ◽  
Caroline Sevin ◽  
...  
Keyword(s):  
Spinal Cord ◽  
Gene Therapy ◽  
Metachromatic Leukodystrophy ◽  
Early Death ◽  
Lysosomal Storage ◽  
Adeno Associated Virus ◽  
Virus Serotype ◽  
Brain And Spinal Cord ◽  
Sulfatide Accumulation

Metachromatic leukodystrophy (MLD) is a lysosomal storage disorder characterized by accumulation of sulfatides in both glial cells and neurons. MLD results from an inherited deficiency of arylsulfatase A (ARSA) and myelin degeneration in the central and peripheral nervous systems. Currently, no effective treatment is available for the most frequent late infantile (LI) form of MLD after symptom onset. The LI form results in rapid neurological degradation and early death. ARSA enzyme must be rapidly and efficiently delivered to brain and spinal cord oligodendrocytes of patients with LI MLD in order to potentially stop the progression of the disease. We previously showed that brain gene therapy with adeno-associated virus serotype rh10 (AAVrh10) driving the expression of human ARSA cDNA alleviated most long-term disease manifestations in MLD mice but was not sufficient in MLD patient to improve disease progression. Herein, we evaluated the short-term effects of intravenous AAVPHP.eB delivery driving the expression of human ARSA cDNA under the control of the cytomegalovirus/b-actin hybrid (CAG) promoter in 6-month-old MLD mice that already show marked sulfatide accumulation and brain pathology. Within 3 months, a single intravenous injection of AAVPHP.eB-hARSA-HA resulted in correction of brain and spinal cord sulfatide storage, and improvement of astrogliosis and microgliosis in brain and spinal cord of treated animals. These results strongly support to consider the use of AAVPHP.eB-hARSA vector for intravenous gene therapy in symptomatic rapidly progressing forms of MLD.

Herpes Simplex Virus Clearance during Purification of a Recombinant Adeno-associated Virus Serotype 1 Vector

2014 ◽  
pp. 150127063140004
Author(s):  
Guo-jie Ye ◽  
Marina M Scotti ◽  
Darby L Thomas ◽  
Lijun Wang ◽  
David R. Knop ◽  
...  
Keyword(s):  
Herpes Simplex Virus ◽  
Herpes Simplex ◽  
Adeno Associated Virus ◽  
Virus Clearance ◽  
Virus Serotype ◽  
Serotype 1 ◽  
Simplex Virus

scholarly journals The 37/67-Kilodalton Laminin Receptor Is a Receptor for Adeno-Associated Virus Serotypes 8, 2, 3, and 9

2006 ◽  
Vol 80 (19) ◽  
pp. 9831-9836 ◽  
Author(s):  
Bassel Akache ◽  
Dirk Grimm ◽  
Kusum Pandey ◽  
Stephen R. Yant ◽  
Hui Xu ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Cultured Cells ◽  
Human Gene ◽  
Laminin Receptor ◽  
Transduction Efficiency ◽  
Adeno Associated Virus ◽  
Virus Serotype ◽  
Tumor Gene

ABSTRACT Adeno-associated virus serotype 8 (AAV8) is currently emerging as a powerful gene transfer vector, owing to its capability to efficiently transduce many different tissues in vivo. While this is believed to be in part due to its ability to uncoat more readily than other AAV serotypes such as AAV2, understanding all the processes behind AAV8 transduction is important for its application and optimal use in human gene therapy. Here, we provide the first report of a cellular receptor for AAV8, the 37/67-kDa laminin receptor (LamR). We document binding of LamR to AAV8 capsid proteins and intact virions in vitro and demonstrate its contribution to AAV8 transduction of cultured cells and mouse liver in vivo. We also show that LamR plays a role in transduction by three other closely related serotypes (AAV2, -3, and -9). Sequence and deletion analysis allowed us to map LamR binding to two protein subdomains predicted to be exposed on the AAV capsid exterior. Use of LamR, which is constitutively expressed in many clinically relevant tissues and is overexpressed in numerous cancers, provides a molecular explanation for AAV8's broad tissue tropism. Along with its robust transduction efficiency, our findings support the continued development of AAV8-based vectors for clinical applications in humans, especially for tumor gene therapy.

scholarly journals Modulation of the liver immune microenvironment by the adeno-associated virus serotype 8 gene therapy vector

2021 ◽  
Vol 20 ◽  
pp. 95-108
Author(s):  
Agostina Carestia ◽  
Seok-Joo Kim ◽  
Franziska Horling ◽  
Hanspeter Rottensteiner ◽  
Christian Lubich ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Immune Microenvironment ◽  
Adeno Associated Virus ◽  
Gene Therapy Vector ◽  
Virus Serotype

scholarly journals Transduction of Nonhuman Primate Brain with Adeno-Associated Virus Serotype 1: Vector Trafficking and Immune Response

2009 ◽  
Vol 20 (3) ◽  
pp. 225-237 ◽  
Author(s):  
Piotr Hadaczek ◽  
John Forsayeth ◽  
Hanna Mirek ◽  
Keith Munson ◽  
John Bringas ◽  
...  
Keyword(s):  
Immune Response ◽  
Nonhuman Primate ◽  
Adeno Associated Virus ◽  
Primate Brain ◽  
Virus Serotype ◽  
Serotype 1
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