Latest developments in the large-scale production of adeno-associated virus vectors in insect cells toward the treatment of neuromuscular diseases
| Autor: | Otto-Wilhelm Merten, Lionel Galibert |
|---|---|
| Přispěvatelé: | Biologie Intégrative et Virologie des Insectes [Univ. de Montpellier II] (BIVI), Institut National de la Recherche Agronomique (INRA)-Université Montpellier 2 - Sciences et Techniques (UM2), Immunologie moléculaire et biothérapies innovantes (IMBI), École Pratique des Hautes Études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL)-Université d'Évry-Val-d'Essonne (UEVE)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Généthon, Généthon-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université d'Évry-Val-d'Essonne (UEVE)-École pratique des hautes études (EPHE), Université Paris sciences et lettres (PSL)-Université Paris sciences et lettres (PSL) |
| Jazyk: | angličtina |
| Rok vydání: | 2011 |
| Předmět: |
Gene Expression Regulation
Viral purification Genetic enhancement viruses [SDV]Life Sciences [q-bio] Genetic Vectors Baculoviridae/*genetics/growth & Sf9 Genetic Therapy/*methods development/immunology Spodoptera Biology Neuromuscular Diseases/genetics/*therapy medicine.disease_cause 03 medical and health sciences Dependovirus/*genetics/immunology 0302 clinical medicine medicine Production (economics) Humans Animals Vector (molecular biology) Viral Adeno-associated virus Gene Ecology Evolution Behavior and Systematics 030304 developmental biology Regulation of gene expression 0303 health sciences Insect cell Genetic Vectors/*genetics/isolation & Gene Transfer Techniques Genetic Therapy Neuromuscular Diseases Dependovirus Virology Spodoptera/*virology Gene Expression Regulation 030220 oncology & carcinogenesis Baculoviridae Biotechnology |
| Zdroj: | J Invertebr Pathol J Invertebr Pathol, 2011, 107 Suppl, pp.S80-93. ⟨10.1016/j.jip.2011.05.008⟩ |
| DOI: | 10.1016/j.jip.2011.05.008⟩ |
| Popis: | International audience; Adeno-associated viral (AAV) vectors are gene vectors of choice for the development of gene therapy treatments for many rare diseases affecting various tissues including retina, central nervous system, liver, and muscle. The AAV based gene therapy approach became conceivable only after the development of easily scalable production systems including the Sf9 cell/baculovirus expression system. Since the establishment of the production of AAV in the Sf9/baculovirus system by the group of Rob Kotin, this new production system has largely been developed for optimizing the large scale production of different serotypes of AAV for preclinical and clinical purposes. Today this manufacturing system allows for the production of purified vector genome (vg) quantities of up to 2 x 10(15) for AAV1 using a 50L reactor and the scale up to larger reactor volumes is paralleled by a corresponding increase in the vector yield. This review presents the principles and achievements of the Sf9/baculovirus system for the production of AAV in comparison to other expression systems based on mammalian cells. In addition, new developments and improvements, which have not yet been implemented at a large scale, and perspectives for further optimization of this production system will be discussed. All of these achievements as well as further process intensifications are urgently needed for the production of clinical doses for the treatment of neuromuscular diseases for which estimated doses of up to 10(14)vg/kg body mass are required. |
| Databáze: | OpenAIRE |
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