PMID- 33108658
OWN - NLM
STAT- MEDLINE
DCOM- 20210326
LR  - 20210326
IS  - 1940-6029 (Electronic)
IS  - 1064-3745 (Linking)
VI  - 2225
DP  - 2021
TI  - Molecular Design and Production of AAV Viral Vectors for Gene Therapy.
PG  - 77-92
LID - 10.1007/978-1-0716-1012-1_5 [doi]
AB  - 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.
FAU - Ridley, Raela B
AU  - Ridley RB
AD  - Department of Ophthalmology, University of Florida College of Medicine, 
      Gainesville, FL, USA.
FAU - Walsh, Erin M
AU  - Walsh EM
AD  - Department of Ophthalmology, University of Florida College of Medicine, 
      Gainesville, FL, USA.
FAU - Ildefonso, Cristhian J
AU  - Ildefonso CJ
AD  - Department of Ophthalmology, University of Florida College of Medicine, 
      Gainesville, FL, USA. ildefons@ufl.edu.
LA  - eng
PT  - Journal Article
PT  - Research Support, N.I.H., Extramural
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - Methods Mol Biol
JT  - Methods in molecular biology (Clifton, N.J.)
JID - 9214969
RN  - 0 (DNA Primers)
RN  - 0 (DNA, Single-Stranded)
SB  - IM
MH  - Cloning, Molecular/*methods
MH  - DNA Primers/chemistry/metabolism
MH  - DNA, Single-Stranded/genetics/metabolism
MH  - Dependovirus/*genetics/metabolism
MH  - Escherichia coli/virology
MH  - Genetic Engineering/*methods
MH  - Genetic Therapy/methods
MH  - Humans
MH  - Plasmids/chemistry/metabolism
MH  - Transduction, Genetic
MH  - *Transgenes
MH  - Viral Genome Packaging/*genetics
OTO - NOTNLM
OT  - AAV
OT  - Cloning
OT  - Gene therapy
OT  - Ligation
OT  - PCR
OT  - Transformation
OT  - Transgene
EDAT- 2020/10/28 06:00
MHDA- 2021/03/27 06:00
CRDT- 2020/10/27 17:40
PHST- 2020/10/27 17:40 [entrez]
PHST- 2020/10/28 06:00 [pubmed]
PHST- 2021/03/27 06:00 [medline]
AID - 10.1007/978-1-0716-1012-1_5 [doi]
PST - ppublish
SO  - Methods Mol Biol. 2021;2225:77-92. doi: 10.1007/978-1-0716-1012-1_5.