Herpes simplex virus co-infection facilitates rolling circle replication of the adeno-associated virus genome. [PDF]
Adeno-associated virus (AAV) genome replication only occurs in the presence of a co-infecting helper virus such as adenovirus type 5 (AdV5) or herpes simplex virus type 1 (HSV-1).
Anouk Lkharrazi +11 more
core +1 more source
Progress in the use of adeno-associated viral vectors for gene therapy [PDF]
The development of safe and efficient gene transfer vectors is crucial for the success of gene therapy trials. A viral vector system promising to meet these requirements is based on the apathogenic adeno-associated virus (AAV-2), a member of the ...
Markus Braun-Falco +5 more
core +1 more source
Adeno-associated Virus DNA Replication [PDF]
ABSTRACT. A model for the replication of adeno-associated virus (AAV) DNA is presented. The AAV genome is a linear single-stranded DNA molecule which contains a 145 nucleotide inverted terminal repetition of which the first 125 nucleotides form a palindromic sequence.
K I, Berns +3 more
openaire +2 more sources
Muscle regeneration affects Adeno Associated Virus 1 mediated transgene transcription
Duchenne muscular dystrophy is a severe neuromuscular disease causing a progressive muscle wasting due to mutations in the DMD gene that lead to the absence of dystrophin protein.
Amédée Mollard +11 more
doaj +1 more source
Process Improvement of Adeno-Associated Virus Production
Adeno-associated viruses (AAVs) have been well characterized and used to deliver therapeutic genes for diseases treatment in clinics and basic research. This study used the triple transient transfection of AAV-DJ/8 as a model expression system to develop
Jia-Shiung Guan +7 more
doaj +1 more source
Efficient delivery of Cre-recombinase to neurons in vivo and stable transduction of neurons using adeno-associated and lentiviral vectors [PDF]
BACKGROUND: Inactivating genes in vivo is an important technique for establishing their function in the adult nervous system. Unfortunately, conventional knockout mice may suffer from several limitations including embryonic or perinatal lethality and the
Hermens, Wim T J M C +39 more
core +1 more source
Adeno-Associated Virus Vectors and Hematology [PDF]
Gene therapists have a special fondness for hematology. Initially, this was due to the many inherited diseases that could potentially be cured by ex vivo genetic modification of hematopoietic stem cells. Once the early murine leukemia virus (MLV) vectors were found to transduce only a small percentage of primate stem cells, the search began for other ...
D W, Russell, M A, Kay
openaire +2 more sources
Expression Profiles of Bovine Adeno-Associated Virus and Avian Adeno-Associated Virus Display Significant Similarity to That of Adeno-Associated Virus Type 5 [PDF]
ABSTRACT We present the first detailed expression profiles of nonprimate-derived adeno-associated viruses, namely, bovine adeno-associated virus (B-AAV) and avian adeno-associated virus (A-AAV), which were obtained after the infection of cell lines derived from their natural hosts.
Jianming, Qiu +2 more
openaire +2 more sources
Gene-edited pseudogene resurrection corrects p47phox-deficient chronic granulomatous disease
: Pseudogenes are duplicated genes with mutations rendering them nonfunctional. For single-gene disorders with homologous pseudogenes, the pseudogene might be a target for genetic correction.
Randall K. Merling +14 more
doaj +1 more source
Adeno-associated virus: fit to serve [PDF]
Adeno-associated virus (AAV) is a helper-dependent parvovirus which has not been linked with human disease. This aspect, in combination with its broad cell and tissue tropism, and limited viral host response has made it an attractive vector system for gene therapy.
Eric Zinn, Luk H Vandenberghe
openaire +2 more sources

