Adeno‐associated virus (AAV)-based gene therapy for glioblastoma
Glioblastoma (GBM) is the most common and malignant Grade IV primary craniocerebral tumor caused by glial cell carcinogenesis with an extremely poor median survival of 12–18 months.
Xin Xu +9 more
doaj +1 more source
Transduction of Salivary Gland Acinar Cells with a Novel AAV Vector 44.9
The loss of salivary gland function caused by radiation therapy of the head and neck or autoimmune disease such as Sjögren’s syndrome is a serious condition that affects a patient’s quality of life. Due to the combined exocrine and endocrine functions of
Giovanni Di Pasquale +14 more
doaj +1 more source
Background Sjögren Syndrome (SS) is a systemic autoimmune disease with a wide spectrum of manifestations that can lead to misdiagnosis. This study describes and compares demographic, clinical, serological, and histopathological data from subjects with SS
Fabíola Reis de Oliveira +6 more
doaj +1 more source
The landscape of viral associations in human cancers [PDF]
Here, as part of the Pan-Cancer Analysis of Whole Genomes (PCAWG) Consortium, for which whole-genome and—for a subset—whole-transcriptome sequencing data from 2,658 cancers across 38 tumor types was aggregated, we systematically investigated potential ...
Alawi, Malik +14 more
core +3 more sources
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 ...
Braun-Falco, M., Buning, H., Hallek, M.
core +1 more source
Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques [PDF]
The feasibility, safety, and efficacy of liver-directed gene transfer was evaluated in 5 male macaques (aged 2.5 to 6.5 years) by using a recombinant adeno-associated viral (rAAV) vector (rAAV-2 CAGG-hFIX) that had previously mediated persistent ...
Davidoff, AM +12 more
core +1 more source
Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. [PDF]
Long-term expression of coagulation factor IX (FIX) has been observed in murine and canine models following administration of recombinant adeno-associated viral (rAAV) vectors into either the portal vein or muscle. These studies were designed to evaluate
Davidoff, A +5 more
core +1 more source
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
Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway. [PDF]
A systematic evaluation of the influence of sex on transduction by recombinant adeno-associated viral vector (rAAV) indicated that transgene expression after liver-targeted delivery of vector particles was between 5- to 13-fold higher in male mice ...
Davidoff, AM +4 more
core +1 more source
Fluorescence Microscopy in Adeno-Associated Virus Research
Research on adeno-associated virus (AAV) and its recombinant vectors as well as on fluorescence microscopy imaging is rapidly progressing driven by clinical applications and new technologies, respectively.
Susanne K. Golm +2 more
doaj +1 more source

