Molecular Design and Production of AAV Viral Vectors for Gene Therapy

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.

Medienart:

E-Artikel

Erscheinungsjahr:

2021

Erschienen:

2021

Enthalten in:

Zur Gesamtaufnahme - volume:2225

Enthalten in:

Methods in molecular biology (Clifton, N.J.) - 2225(2021) vom: 27., Seite 77-92

Sprache:

Englisch

Beteiligte Personen:

Ridley, Raela B [VerfasserIn]
Walsh, Erin M [VerfasserIn]
Ildefonso, Cristhian J [VerfasserIn]

Links:

Volltext

Themen:

AAV
Cloning
DNA, Single-Stranded
DNA Primers
Gene therapy
Journal Article
Ligation
PCR
Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't
Transformation
Transgene

Anmerkungen:

Date Completed 26.03.2021

Date Revised 26.03.2021

published: Print

Citation Status MEDLINE

doi:

10.1007/978-1-0716-1012-1_5

funding:

Förderinstitution / Projekttitel:

PPN (Katalog-ID):

NLM316773271