Molecular Design and Production of AAV Viral Vectors for Gene Therapy.
Methods Mol Biol
; 2225: 77-92, 2021.
Article
em En
| MEDLINE
| ID: mdl-33108658
ABSTRACT
Adeno-associated virus (AAV) is a helper-dependent single-stranded DNA parvovirus. Over the years, AAV has become the vector of choice in the gene therapy field due to its safety profile and low immunogenicity. With a carrying capacity of 4.2 kbp, these vectors have demonstrated their clinical value, especially in the field of ophthalmology. Herein we describe methods for the molecular design and packaging of AAV viral vectors. These methods apply to the design of single-stranded or self-complementary AAV vectors.
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Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Engenharia Genética
/
Clonagem Molecular
/
Dependovirus
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Transgenes
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Empacotamento do Genoma Viral
Limite:
Humans
Idioma:
En
Ano de publicação:
2021
Tipo de documento:
Article